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The search results on this publication page are automated on a monthly schedule based on acknowledgement of NIH Common Fund award numbers and intramural awards. Therefore, this list is not an exhaustive or error-free account of the program’s publications.

Title Authors Journal PubMedID Publication Date
Production of High-Titer Retrovirus and Lentivirus Vectors. Sena-Esteves, Miguel; Gao, Guangping Cold Spring Harbor protocols. 29610362 2018 04 02;
Titration of Lentivirus Vectors. Sena-Esteves, Miguel; Gao, Guangping Cold Spring Harbor protocols. 29610363 2018 04 02;
Monitoring Lentivirus Vector Stocks for Replication-Competent Viruses. Sena-Esteves, Miguel; Gao, Guangping Cold Spring Harbor protocols. 29610364 2018 04 02;
H3Africa: current perspectives. Mulder, Nicola; Abimiku, Alash'le; Adebamowo, Sally N; de Vries, Jantina; Matimba, Alice; Olowoyo, Paul; Ramsay, Michele; Skelton, Michelle; Stein, Dan J Pharmacogenomics and personalized medicine. 29692621 2018;
Clinical Evaluations of Toxicity and Efficacy of Nanoparticle-Mediated Gene Therapy. Liang, Xiao; Liu, Li; Wei, Yu-Quan; Gao, Guang-Ping; Wei, Xia-Wei Human gene therapy. 29893153 2018 11;
Cas9-mediated allelic exchange repairs compound heterozygous recessive mutations in mice. Wang, Dan; Li, Jia; Song, Chun-Qing; Tran, Karen; Mou, Haiwei; Wu, Pei-Hsuan; Tai, Phillip W L; Mendonca, Craig A; Ren, Lingzhi; Wang, Blake Y; Su, Qin; Gessler, Dominic J; Zamore, Phillip D; Xue, Wen; Gao, Guangping Nature biotechnology. 30102296 2018 Oct;
Tunability of Biodegradable Poly(amine- co-ester) Polymers for Customized Nucleic Acid Delivery and Other Biomedical Applications. Kauffman, Amy C; Piotrowski-Daspit, Alexandra S; Nakazawa, Kay H; Jiang, Yuhang; Datye, Amit; Saltzman, W Mark Biomacromolecules 30110158 2018 09 10
The temporal landscape of recursive splicing during Pol II transcription elongation in human cells. Zhang, Xiao-Ou; Fu, Yu; Mou, Haiwei; Xue, Wen; Weng, Zhiping PLoS genetics. 30148885 2018 08;
All-in-one adeno-associated virus delivery and genome editing by Neisseria meningitidis Cas9 in vivo. Ibraheim, Raed; Song, Chun-Qing; Mir, Aamir; Amrani, Nadia; Xue, Wen; Sontheimer, Erik J Genome biology. 30231914 2018 Sep 19;
Defining CRISPR-Cas9 genome-wide nuclease activities with CIRCLE-seq. Lazzarotto, Cicera R; Nguyen, Nhu T; Tang, Xing; Malagon-Lopez, Jose; Guo, Jimmy A; Aryee, Martin J; Joung, J Keith; Tsai, Shengdar Q Nature protocols. 30341435 2018 11;
Intrathecal Adeno-Associated Viral Vector-Mediated Gene Delivery for Adrenomyeloneuropathy. Gong, Yi; Berenson, Anna; Laheji, Fiza; Gao, Guangping; Wang, Dan; Ng, Carrie; Volak, Adrienn; Kok, Rene; Kreouzis, Vasileios; Dijkstra, Inge M; Kemp, Stephan; Maguire, Casey A; Eichler, Florian Human gene therapy. 30358470 2019 May;
Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer. Wang, Dan; Zhong, Li; Li, Mengxin; Li, Jia; Tran, Karen; Ren, Lingzhi; He, Ran; Xie, Jun; Moser, Richard P; Fraser, Cara; Kuchel, Tim; Sena-Esteves, Miguel; Flotte, Terence R; Aronin, Neil; Gao, Guangping Molecular therapy. Methods & clinical development. 30397628 2018 Dec 14;
Understanding and repurposing CRISPR-mediated alternative splicing. Smith, Jordan L; Mou, Haiwei; Xue, Wen Genome biology. 30400804 2018 11 06;
MicroRNA-96 Promotes Schistosomiasis Hepatic Fibrosis in Mice by Suppressing Smad7. Luo, Xufeng; Zhang, Dongmei; Xie, Jun; Su, Qin; He, Xing; Bai, Ruipu; Gao, Guangping; Pan, Weiqing Molecular therapy. Methods & clinical development. 30406154 2018 Dec 14;
Targeting fidelity of adenine and cytosine base editors in mouse embryos. Lee, Hye Kyung; Willi, Michaela; Miller, Shannon M; Kim, Sojung; Liu, Chengyu; Liu, David R; Hennighausen, Lothar Nature communications. 30442934 2018 Nov 15;
Intracellular Delivery of His-Tagged Genome-Editing Proteins Enabled by Nitrilotriacetic Acid-Containing Lipidoid Nanoparticles. Li, Yamin; Li, Alice Chukun; Xu, Qiaobing Advanced healthcare materials. 30565897 2019 Mar;
Illuminating the genome-wide activity of genome editors for safe and effective therapeutics. Cheng, Yong; Tsai, Shengdar Q Genome biology. 30577870 2018 12 22;
A Compact, High-Accuracy Cas9 with a Dinucleotide PAM for In Vivo Genome Editing. Edraki, Alireza; Mir, Aamir; Ibraheim, Raed; Gainetdinov, Ildar; Yoon, Yeonsoo; Song, Chun-Qing; Cao, Yueying; Gallant, Judith; Xue, Wen; Rivera-Pérez, Jaime A; Sontheimer, Erik J Molecular cell. 30581144 2018 Dec 18;
Integrating Combinatorial Lipid Nanoparticle and Chemically Modified Protein for Intracellular Delivery and Genome Editing. Chang, Jin; Chen, Xianghan; Glass, Zachary; Gao, Feng; Mao, Lanqun; Wang, Ming; Xu, Qiaobing Accounts of chemical research. 30586281 2019 Mar 19;
Enhancing the In Vitro and In Vivo Stabilities of Polymeric Nucleic Acid Delivery Nanosystems. Wang, Yuyuan; Ye, Mingzhou; Xie, Ruosen; Gong, Shaoqin Bioconjugate chemistry. 30592619 2019 02 20;
Anti-drug Antibody Responses Impair Prophylaxis Mediated by AAV-Delivered HIV-1 Broadly Neutralizing Antibodies. Gardner, Matthew R; Fetzer, Ina; Kattenhorn, Lisa M; Davis-Gardner, Meredith E; Zhou, Amber S; Alfant, Barnett; Weber, Jesse A; Kondur, Hema R; Martinez-Navio, Jose M; Fuchs, Sebastian P; Desrosiers, Ronald C; Gao, Guangping; Lifson, Jeffrey D; Farzan, Mi Molecular therapy : the journal of the American Society of Gene Therapy. 30704961 2019 03 06;
Adeno-associated virus vector as a platform for gene therapy delivery. Wang, Dan; Tai, Phillip W L; Gao, Guangping Nature reviews. Drug discovery. 30710128 2019 05;
Simultaneous targeting of linked loci in mouse embryos using base editing. Lee, Hye Kyung; Willi, Michaela; Smith, Harold E; Miller, Shannon M; Liu, David R; Liu, Chengyu; Hennighausen, Lothar Scientific reports. 30733567 2019 Feb 07;
Quantitative and Digital Droplet-Based AAV Genome Titration. Sanmiguel, Julio; Gao, Guangping; Vandenberghe, Luk H Methods in molecular biology (Clifton, N.J.). 30783968 2019;
Intravenous Infusion of AAV for Widespread Gene Delivery to the Nervous System. Gessler, Dominic J; Tai, Phillip W L; Li, Jia; Gao, Guangping Methods in molecular biology (Clifton, N.J.). 30783972 2019;
Kidney organoids in translational medicine: Disease modeling and regenerative medicine. Miyoshi, Tomoya; Hiratsuka, Ken; Saiz, Edgar Garcia; Morizane, Ryuji Developmental dynamics : an official publication of the American Association of Anatomists 30843293 2020 01
Adeno-Associated Virus Delivery of Anti-HIV Monoclonal Antibodies Can Drive Long-Term Virologic Suppression. Martinez-Navio, José M; Fuchs, Sebastian P; Pantry, Shara N; Lauer, William A; Duggan, Natasha N; Keele, Brandon F; Rakasz, Eva G; Gao, Guangping; Lifson, Jeffrey D; Desrosiers, Ronald C Immunity. 30850342 2019 Mar 19;
Enhanced Cas12a editing in mammalian cells and zebrafish. Liu, Pengpeng; Luk, Kevin; Shin, Masahiro; Idrizi, Feston; Kwok, Samantha; Roscoe, Benjamin; Mintzer, Esther; Suresh, Sneha; Morrison, Kyle; Frazão, Josias B; Bolukbasi, Mehmet Fatih; Ponnienselvan, Karthikeyan; Luban, Jeremy; Zhu, Lihua Julie; Lawson, Nathan D; Wolfe, Scot A Nucleic acids research. 30892626 2019 Mar 20;
An orange calcium-modulated bioluminescent indicator for non-invasive activity imaging. Oh, Younghee; Park, Yunhee; Cho, Julia H; Wu, Haodi; Paulk, Nicole K; Liu, Lan Xiang; Kim, Namdoo; Kay, Mark A; Wu, Joseph C; Lin, Michael Z Nature chemical biology. 30936501 2019 05;
CRISPR-SONIC: targeted somatic oncogene knock-in enables rapid in vivo cancer modeling. Mou, Haiwei; Ozata, Deniz M; Smith, Jordan L; Sheel, Ankur; Kwan, Suet-Yan; Hough, Soren; Kucukural, Alper; Kennedy, Zachary; Cao, Yueying; Xue, Wen Genome medicine. 30987660 2019 04 16;
Unbiased detection of CRISPR off-targets in vivo using DISCOVER-Seq. Wienert, Beeke; Wyman, Stacia K; Richardson, Christopher D; Yeh, Charles D; Akcakaya, Pinar; Porritt, Michelle J; Morlock, Michaela; Vu, Jonathan T; Kazane, Katelynn R; Watry, Hannah L; Judge, Luke M; Conklin, Bruce R; Maresca, Marcello; Corn, Jacob E Science (New York, N.Y.). 31000663 2019 04 19;
Nucleic Acid Therapeutics for Neurological Diseases. Watts, Jonathan K; Brown, Robert H; Khvorova, Anastasia Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics. 31037650 2019 04;
Construction of Recombinant Adenovirus Genomes by Direct Cloning. Zhou, Xiangyang; Sena-Esteves, Miguel; Gao, Guangping Cold Spring Harbor protocols. 31043558 2019 05 01;
Release of the Cloned Recombinant Adenovirus Genome for Rescue and Expansion. Su, Qin; Sena-Esteves, Miguel; Gao, Guangping Cold Spring Harbor protocols. 31043559 2019 05 01;
Purification of the Recombinant Adenovirus by Cesium Chloride Gradient Centrifugation. Su, Qin; Sena-Esteves, Miguel; Gao, Guangping Cold Spring Harbor protocols. 31043560 2019 05 01;
High-Throughput Quantification of In Vivo Adeno-Associated Virus Transduction with Barcoded Non-Coding RNAs. Xu, Meiyu; Li, Jia; Xie, Jun; He, Ran; Su, Qin; Gao, Guangping; Tai, Phillip W L Human gene therapy. 31072208 2019 Jun 17;
Analysis and minimization of cellular RNA editing by DNA adenine base editors. Rees, Holly A; Wilson, Christopher; Doman, Jordan L; Liu, David R Science advances. 31086823 2019 May;
Development of hRad51-Cas9 nickase fusions that mediate HDR without double-stranded breaks. Rees, Holly A; Yeh, Wei-Hsi; Liu, David R Nature communications. 31101808 2019 05 17;
Circularly permuted and PAM-modified Cas9 variants broaden the targeting scope of base editors. Huang, Tony P; Zhao, Kevin T; Miller, Shannon M; Gaudelli, Nicole M; Oakes, Benjamin L; Fellmann, Christof; Savage, David F; Liu, David R Nature biotechnology. 31110355 2019 Jun;
Circumventing cellular immunity by miR142-mediated regulation sufficiently supports rAAV-delivered OVA expression without activating humoral immunity. Xiao, Yuanyuan; Muhuri, Manish; Li, Shaoyong; Qin, Wanru; Xu, Guangchao; Luo, Li; Li, Jia; Letizia, Alexander J; Wang, Sean K; Chan, Ying Kai; Wang, Chunmei; Fuchs, Sebastian P; Wang, Dan; Su, Qin; Nahid, M Abu; Church, George M; Farzan, Michael; Yang, Li; Wei, Yuquan; Desrosiers, Ronald C; Mueller, Christian; Tai, Phillip Wl; Gao, Guangping JCI insight. 31112525 2019 May 21;
Depletion of TRRAP Induces p53-Independent Senescence in Liver Cancer by Down-Regulating Mitotic Genes. Kwan, Suet-Yan; Sheel, Ankur; Song, Chun-Qing; Zhang, Xiao-Ou; Jiang, Tingting; Dang, Hien; Cao, Yueying; Ozata, Deniz M; Mou, Haiwei; Yin, Hao; Weng, Zhiping; Wang, Xin Wei; Xue, Wen Hepatology (Baltimore, Md.). 31188495 2019 Jun 12;
Fast and Efficient CRISPR/Cas9 Genome Editing In Vivo Enabled by Bioreducible Lipid and Messenger RNA Nanoparticles. Liu, Ji; Chang, Jin; Jiang, Ying; Meng, Xiandi; Sun, Tianmeng; Mao, Lanqun; Xu, Qiaobing; Wang, Ming Advanced materials (Deerfield Beach, Fla.). 31215123 2019 Jun 19;
An anionic human protein mediates cationic liposome delivery of genome editing proteins into mammalian cells. Kim, Y Bill; Zhao, Kevin T; Thompson, David B; Liu, David R Nature communications. 31266953 2019 07 02;
Bone-targeting AAV-mediated silencing of Schnurri-3 prevents bone loss in osteoporosis. Yang, Yeon-Suk; Xie, Jun; Wang, Dan; Kim, Jung-Min; Tai, Phillip W L; Gravallese, Ellen; Gao, Guangping; Shim, Jae-Hyuck Nature communications. 31273195 2019 07 04;
Continuous evolution of base editors with expanded target compatibility and improved activity. Thuronyi, Benjamin W; Koblan, Luke W; Levy, Jonathan M; Yeh, Wei-Hsi; Zheng, Christine; Newby, Gregory A; Wilson, Christopher; Bhaumik, Mantu; Shubina-Oleinik, Olga; Holt, Jeffrey R; Liu, David R Nature biotechnology. 31332326 2019 Jul 22;
Nonviral Nanoparticles for CRISPR-Based Genome Editing: Is It Just a Simple Adaption of What Have Been Developed for Nucleic Acid Delivery? Qiu, Min; Glass, Zachary; Xu, Qiaobing Biomacromolecules. 31342740 2019 09 09;
Mapping cis-regulatory chromatin contacts in neural cells links neuropsychiatric disorder risk variants to target genes. Song, Michael; Yang, Xiaoyu; Ren, Xingjie; Maliskova, Lenka; Li, Bingkun; Jones, Ian R; Wang, Chao; Jacob, Fadi; Wu, Kenneth; Traglia, Michela; Tam, Tsz Wai; Jamieson, Kirsty; Lu, Si-Yao; Ming, Guo-Li; Li, Yun; Yao, Jun; Weiss, Lauren A; Dixon, Jesse R; J Nature genetics. 31367015 2019 08;
Measuring the Infectious Titer of Recombinant Adenovirus Using Tissue Culture Infection Dose 50% (TCID50) End-Point Dilution and Quantitative Polymerase Chain Reaction (qPCR). Lock, Martin; Korn, Michael; Wilson, James; Sena-Esteves, Miguel; Gao, Guangping Cold Spring Harbor protocols. 31371467 2019 Aug 01;
Preparation of a DNA Standard for Quantitative Polymerase Chain Reaction (qPCR). Lock, Martin; Korn, Michael; Wilson, James; Sena-Esteves, Miguel; Gao, Guangping Cold Spring Harbor protocols. 31371468 2019 Aug 01;
Base Editor Correction of COL7A1 in Recessive Dystrophic Epidermolysis Bullosa Patient-Derived Fibroblasts and iPSCs. Osborn, Mark J; Newby, Gregory A; McElroy, Amber N; Knipping, Friederike; Nielsen, Sarah C; Riddle, Megan J; Xia, Lily; Chen, Weili; Eide, Cindy R; Webber, Beau R; Wandall, Hans H; Dabelsteen, Sally; Blazar, Bruce R; Liu, David R; Tolar, Jakub The Journal of investigative dermatology. 31437443 2020 Feb;
Tissue-restricted genome editing in vivo specified by microRNA-repressible anti-CRISPR proteins. Lee, Jooyoung; Mou, Haiwei; Ibraheim, Raed; Liang, Shun-Qing; Liu, Pengpeng; Xue, Wen; Sontheimer, Erik J RNA (New York, N.Y.). 31439808 2019 11;
Noninvasive imaging of renal urea handling by CEST-MRI. Shin, Soo Hyun; Wendland, Michael F; Zhang, Brandon; Tran, An; Tang, Albert; Vandsburger, Moriel H Magnetic resonance in medicine 31483529 2020 03
A biodegradable nanocapsule delivers a Cas9 ribonucleoprotein complex for in vivo genome editing. Chen, Guojun; Abdeen, Amr A; Wang, Yuyuan; Shahi, Pawan K; Robertson, Samantha; Xie, Ruosen; Suzuki, Masatoshi; Pattnaik, Bikash R; Saha, Krishanu; Gong, Shaoqin Nature nanotechnology. 31501532 2019 Oct;
Blueprint for building a biorepository in a resource-limited setting that follows international best practices. Abimiku, Alash'le G; Croxton, Talishea; Ozumba, Petronilla J; Agala, Ndidi; Balogun, Olasinbo; Jonathan, Emmanuel; Onyemata, Enzenwa; Ndifon, Kachimi; Nadoma, Sunji; Anazodo, Thankgod; Peters, Sam; Beiswanger, Christine M African journal of laboratory medicine. 31534913 2019;
CRISPR genome editing in stem cells turns to gold. Asokan, Aravind Nature materials 31537943 2019 10
Search-and-replace genome editing without double-strand breaks or donor DNA. Anzalone, Andrew V; Randolph, Peyton B; Davis, Jessie R; Sousa, Alexander A; Koblan, Luke W; Levy, Jonathan M; Chen, Peter J; Wilson, Christopher; Newby, Gregory A; Raguram, Aditya; Liu, David R Nature. 31634902 2019 12;
Optimizing biodegradable nanoparticle size for tissue-specific delivery. Mandl, Hanna K; Quijano, Elias; Suh, Hee Won; Sparago, Emily; Oeck, Sebastian; Grun, Molly; Glazer, Peter M; Saltzman, W Mark Journal of controlled release : official journal of the Controlled Release Society. 31654688 2019 Nov 28;
Engineered amphiphilic peptides enable delivery of proteins and CRISPR-associated nucleases to airway epithelia. Krishnamurthy, Sateesh; Wohlford-Lenane, Christine; Kandimalla, Suhas; Sartre, Gilles; Meyerholz, David K; Théberge, Vanessa; Hallée, Stéphanie; Duperré, Anne-Marie; Del'Guidice, Thomas; Lepetit-Stoffaes, Jean-Pascal; Barbeau, Xavier; Guay, David; McCray Nature communications. 31659165 2019 10 28;
Structures of Neisseria meningitidis Cas9 Complexes in Catalytically Poised and Anti-CRISPR-Inhibited States. Sun, Wei; Yang, Jing; Cheng, Zhi; Amrani, Nadia; Liu, Chao; Wang, Kangkang; Ibraheim, Raed; Edraki, Alireza; Huang, Xue; Wang, Min; Wang, Jiuyu; Liu, Liang; Sheng, Gang; Yang, Yanhua; Lou, Jizhong; Sontheimer, Erik J; Wang, Yanli Molecular cell. 31668930 2019 12 19;
Genome editing of HBG1 and HBG2 to induce fetal hemoglobin. Métais, Jean-Yves; Doerfler, Phillip A; Mayuranathan, Thiyagaraj; Bauer, Daniel E; Fowler, Stephanie C; Hsieh, Matthew M; Katta, Varun; Keriwala, Sagar; Lazzarotto, Cicera R; Luk, Kevin; Neel, Michael D; Perry, S Scott; Peters, Samuel T; Porter, Shaina N; Blood advances. 31698466 2019 11 12;
Delivering genes across the blood-brain barrier: LY6A, a novel cellular receptor for AAV-PHP.B capsids. Huang, Qin; Chan, Ken Y; Tobey, Isabelle G; Chan, Yujia Alina; Poterba, Tim; Boutros, Christine L; Balazs, Alejandro B; Daneman, Richard; Bloom, Jonathan M; Seed, Cotton; Deverman, Benjamin E PloS one. 31725765 2019;
Gene Delivery to Nonhuman Primate Preimplantation Embryos Using Recombinant Adeno-Associated Virus. Wang, Dan; Niu, Yuyu; Ren, Lingzhi; Kang, Yu; Tai, Phillip W L; Si, Chenyang; Mendonca, Craig A; Ma, Hong; Gao, Guangping; Ji, Weizhi Advanced science (Weinheim, Baden-Wurttemberg, Germany). 31728271 2019 Nov 06;
Adenine base editing in an adult mouse model of tyrosinaemia. Song, Chun-Qing; Jiang, Tingting; Richter, Michelle; Rhym, Luke H; Koblan, Luke W; Zafra, Maria Paz; Schatoff, Emma M; Doman, Jordan L; Cao, Yueying; Dow, Lukas E; Zhu, Lihua Julie; Anderson, Daniel G; Liu, David R; Yin, Hao; Xue, Wen Nature biomedical engineering. 31740768 2020 Jan;
Fast-Seq: A Simple Method for Rapid and Inexpensive Validation of Packaged Single-Stranded Adeno-Associated Viral Genomes in Academic Settings. Maynard, Lucy H; Smith, Olivia; Tilmans, Nicolas P; Tham, Eleonore; Hosseinzadeh, Shayan; Tan, Weilun; Leenay, Ryan; May, Andrew P; Paulk, Nicole K Human gene therapy methods. 31855083 2019 12;
Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses. Levy, Jonathan M; Yeh, Wei-Hsi; Pendse, Nachiket; Davis, Jessie R; Hennessey, Erin; Butcher, Rossano; Koblan, Luke W; Comander, Jason; Liu, Qin; Liu, David R Nature biomedical engineering. 31937940 2020 Jan;
Ex vivo cell-based CRISPR/Cas9 genome editing for therapeutic applications. Li, Yamin; Glass, Zachary; Huang, Mingqian; Chen, Zheng-Yi; Xu, Qiaobing Biomaterials. 31945616 2020 Mar;
Detection of Extracellular Vesicle RNA Using Molecular Beacons. Oliveira Jr, Getulio Pereira de; Zigon, Eric; Rogers, Gaenna; Davodian, Danny; Lu, Shulin; Jovanovic-Talisman, Tijana; Jones, Jennifer; Tigges, John; Tyagi, Sanjay; Ghiran, Ionita Calin iScience. 31958756 2020 Jan 24;
From GWAS Association to Function: Candidate Gene Screening Within Insulin Resistance-Associated Genomic Loci Using a Preadipocyte Differentiation Model. Ladha, Feria A; Stitzel, Michael L; Hinson, J Travis Circulation research. 31999535 2020 Jan 31;
Quantitating Endosomal Escape of a Library of Polymers for mRNA Delivery. Jiang, Yuhang; Lu, Qiao; Wang, Yongheng; Xu, Emily; Ho, Alison; Singh, Priya; Wang, Yifei; Jiang, Zhaozhong; Yang, Fan; Tietjen, Gregory T; Cresswell, Peter; Saltzman, W Mark Nano letters. 32003222 2020 Feb 12;
High-throughput quantitative microscopy-based half-life measurements of intravenously injected agents. Bracaglia, Laura G; Piotrowski-Daspit, Alexandra S; Lin, Chun-Yu; Moscato, Zoe M; Wang, Yongheng; Tietjen, Gregory T; Saltzman, W Mark Proceedings of the National Academy of Sciences of the United States of America. 32015123 2020 Feb 18;
Evaluation and minimization of Cas9-independent off-target DNA editing by cytosine base editors. Doman, Jordan L; Raguram, Aditya; Newby, Gregory A; Liu, David R Nature biotechnology. 32042165 2020 Feb 10;
Continuous evolution of SpCas9 variants compatible with non-G PAMs. Miller, Shannon M; Wang, Tina; Randolph, Peyton B; Arbab, Mandana; Shen, Max W; Huang, Tony P; Matuszek, Zaneta; Newby, Gregory A; Rees, Holly A; Liu, David R Nature biotechnology. 32042170 2020 Apr;
Peptide Nucleic Acids and Gene Editing: Perspectives on Structure and Repair. Economos, Nicholas G; Oyaghire, Stanley; Quijano, Elias; Ricciardi, Adele S; Saltzman, W Mark; Glazer, Peter M Molecules (Basel, Switzerland). 32046275 2020 Feb 08;
Clades of huge phages from across Earth's ecosystems. Al-Shayeb, Basem; Sachdeva, Rohan; Chen, Lin-Xing; Ward, Fred; Munk, Patrick; Devoto, Audra; Castelle, Cindy J; Olm, Matthew R; Bouma-Gregson, Keith; Amano, Yuki; He, Christine; Méheust, Raphaël; Brooks, Brandon; Thomas, Alex; Lavy, Adi; Matheus-Carnevali Nature. 32051592 2020 02;
The promise and challenge of therapeutic genome editing. Doudna, Jennifer A Nature 32051598 2020 02
Site-Specific Post-translational Surface Modification of Adeno-Associated Virus Vectors Using Leucine Zippers. Thadani, Nicole N; Yang, Joanna; Moyo, Buhle; Lee, Ciaran M; Chen, Maria Y; Bao, Gang; Suh, Junghae ACS synthetic biology. 32068391 2020 Mar 20;
Poly(Lactic-co-Glycolic Acid) Nanoparticle Delivery of Peptide Nucleic Acids In Vivo. Oyaghire, Stanley N; Quijano, Elias; Piotrowski-Daspit, Alexandra S; Saltzman, W Mark; Glazer, Peter M Methods in molecular biology (Clifton, N.J.). 32088877 2020;
A New Gorilla Adenoviral Vector with Natural Lung Tropism Avoids Liver Toxicity and Is Amenable to Capsid Engineering and Vector Retargeting. Lu, Zhi Hong; Dmitriev, Igor P; Brough, Douglas E; Kashentseva, Elena A; Li, Jie; Curiel, David T Journal of virology 32102889 2020 05 04
Can we mimic skeletal muscles for novel drug discovery? Broer, Torie; Khodabukus, Alastair; Bursac, Nenad Expert opinion on drug discovery. 32122176 2020 Mar 03;
Targeted Delivery of Secretory Promelittin via Novel Poly(lactone-co-?-amino ester) Nanoparticles for Treatment of Breast Cancer Brain Metastases. Zhou, Yu; Zhang, Shenqi; Chen, Zeming; Bao, Youmei; Chen, Ann T; Sheu, Wendy C; Liu, Fuyao; Jiang, Zhaozhong; Zhou, Jiangbing Advanced science (Weinheim, Baden-Wurttemberg, Germany). 32154067 2020 Mar;
CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors. Wang, Dan; Zhang, Feng; Gao, Guangping Cell. 32243786 2020 Apr 02;
Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases. Wu, Shao-Shuai; Li, Qing-Cui; Yin, Chang-Qing; Xue, Wen; Song, Chun-Qing Theranostics. 32292501 2020;
Tissue-Engineered Human Myobundle System as a Platform for Evaluation of Skeletal Muscle Injury Biomarkers. Khodabukus, Alastair; Kaza, Amulya; Wang, Jason; Prabhu, Neel; Goldstein, Richard; Vaidya, Vishal S; Bursac, Nenad Toxicological sciences : an official journal of the Society of Toxicology. 32294208 2020 Jul 01;
Small-Molecule PAPD5 Inhibitors Restore Telomerase Activity in Patient Stem Cells. Nagpal, Neha; Wang, Jianing; Zeng, Jing; Lo, Emily; Moon, Diane H; Luk, Kevin; Braun, Roman O; Burroughs, Lauri M; Keel, Sioban B; Reilly, Christopher; Lindsley, R Coleman; Wolfe, Scot A; Tai, Albert K; Cahan, Patrick; Bauer, Daniel E; Fong, Yick W; Agarwal, Suneet Cell stem cell. 32320679 2020 Jun 04;
Myogenic Progenitor Cell Lineage Specification by CRISPR/Cas9-Based Transcriptional Activators. Kwon, Jennifer B; Vankara, Ashish; Ettyreddy, Adarsh R; Bohning, Joel D; Gersbach, Charles A Stem cell reports. 32330446 2020 05 12;
Chemical modifications of adenine base editor mRNA and guide RNA expand its application scope. Jiang, Tingting; Henderson, Jordana M; Coote, Kevin; Cheng, Yi; Valley, Hillary C; Zhang, Xiao-Ou; Wang, Qin; Rhym, Luke H; Cao, Yueying; Newby, Gregory A; Bihler, Hermann; Mense, Martin; Weng, Zhiping; Anderson, Daniel G; McCaffrey, Anton P; Liu, David R; Xue, Wen Nature communications. 32332735 2020 Apr 24;
AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9. Li, Ang; Tanner, Mark R; Lee, Ciaran M; Hurley, Ayrea E; De Giorgi, Marco; Jarrett, Kelsey E; Davis, Timothy H; Doerfler, Alexandria M; Bao, Gang; Beeton, Christine; Lagor, William R Molecular therapy : the journal of the American Society of Gene Therapy. 32348718 2020 Jun 03;
Positron emission tomography imaging of novel AAV capsids maps rapid brain accumulation. Seo, Jai Woong; Ingham, Elizabeth S; Mahakian, Lisa; Tumbale, Spencer; Wu, Bo; Aghevlian, Sadaf; Shams, Shahin; Baikoghli, Mo; Jain, Poorva; Ding, Xiaozhe; Goeden, Nick; Dobreva, Tatyana; Flytzanis, Nicholas C; Chavez, Michael; Singhal, Kratika; Leib, Rya Nature communications. 32355221 2020 04 30;
Finding order in slow axonal transport. Roy, Subhojit Current opinion in neurobiology. 32361600 2020 08;
Once upon a dish: engineering multicellular systems. Haase, Kristina; Freedman, Benjamin S Development (Cambridge, England). 32366376 2020 May 04;
A pH-responsive silica-metal-organic framework hybrid nanoparticle for the delivery of hydrophilic drugs, nucleic acids, and CRISPR-Cas9 genome-editing machineries. Wang, Yuyuan; Shahi, Pawan K; Xie, Ruosen; Zhang, Huilong; Abdeen, Amr A; Yodsanit, Nisakorn; Ma, Zhenqiang; Saha, Krishanu; Pattnaik, Bikash R; Gong, Shaoqin Journal of controlled release : official journal of the Controlled Release Society. 32380204 2020 May 05;
Advances in genome editing for genetic hearing loss. Ding, Ning; Lee, Sangsin; Lieber-Kotz, Matan; Yang, Jie; Gao, Xue Advanced drug delivery reviews. 32387678 2020 May 07;
Prime genome editing in rice and wheat. Lin, Qiupeng; Zong, Yuan; Xue, Chenxiao; Wang, Shengxing; Jin, Shuai; Zhu, Zixu; Wang, Yanpeng; Anzalone, Andrew V; Raguram, Aditya; Doman, Jordan L; Liu, David R; Gao, Caixia Nature biotechnology. 32393904 2020 05;
Effect of luminal flow on doming of mpkCCD cells in a 3D perfusable kidney cortical collecting duct model. Rein, Joshua L; Heja, Szilvia; Flores, Daniel; Carrisoza-Gaytán, Rolando; Lin, Neil Y C; Homan, Kimberly A; Lewis, Jennifer A; Satlin, Lisa M American journal of physiology. Cell physiology 32401606 2020 07 01
Transcriptomic and Proteostasis Networks of CFTR and the Development of Small Molecule Modulators for the Treatment of Cystic Fibrosis Lung Disease. Strub, Matthew D; McCray Jr, Paul B Genes 32414011 2020 05 13
Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity. Richter, Michelle F; Zhao, Kevin T; Eton, Elliot; Lapinaite, Audrone; Newby, Gregory A; Thuronyi, Benjamin W; Wilson, Christopher; Koblan, Luke W; Zeng, Jing; Bauer, Daniel E; Doudna, Jennifer A; Liu, David R Nature biotechnology. 32433547 2020 Mar 16;
Protein and mRNA Delivery Enabled by Cholesteryl-Based Biodegradable Lipidoid Nanoparticles. Li, Yamin; Jarvis, Rachel; Zhu, Kuixin; Glass, Zachary; Ogurlu, Roza; Gao, Peiyang; Li, Peixuan; Chen, Jinjin; Yu, Yingjie; Yang, Yongjie; Xu, Qiaobing Angewandte Chemie (International ed. in English). 32438474 2020 May 21;
The developing toolkit of continuous directed evolution. Morrison, Mary S; Podracky, Christopher J; Liu, David R Nature chemical biology. 32444838 2020 Jun;
YAP1 withdrawal in hepatoblastoma drives therapeutic differentiation of tumor cells to functional hepatocyte-like cells. Smith, Jordan L; Rodríguez, Tomás C; Mou, Haiwei; Kwan, Suet-Yan; Pratt, Henry; Zhang, Xiao-Ou; Cao, Yueying; Liang, Shunqing; Ozata, Deniz M; Yu, Tianxiong; Yin, Qiangzong; Hazeltine, Max; Weng, Zhiping; Sontheimer, Erik J; Xue, Wen Hepatology (Baltimore, Md.). 32452550 2020 May 26;
In vivo base editing restores sensory transduction and transiently improves auditory function in a mouse model of recessive deafness. Yeh, Wei-Hsi; Shubina-Oleinik, Olga; Levy, Jonathan M; Pan, Bifeng; Newby, Gregory A; Wornow, Michael; Burt, Rachel; Chen, Jonathan C; Holt, Jeffrey R; Liu, David R Science translational medicine 32493795 2020 06 03
Combinatorial Library of Cyclic Benzylidene Acetal-Containing pH-Responsive Lipidoid Nanoparticles for Intracellular mRNA Delivery. Li, Yamin; Li, Raissa; Chakraborty, Anirban; Ogurlu, Roza; Zhao, Xuewei; Chen, Jinjin; Xu, Qiaobing Bioconjugate chemistry. 32520527 2020 07 15;
Defining Transcription Regulatory Elements in the Human Frataxin Gene: Implications for Gene Therapy. Li, Jixue; Li, Yanjie; Wang, Jun; Gonzalez, Trevor J; Asokan, Aravind; Napierala, Jill S; Napierala, Marek Human gene therapy 32527155 2020 08
Determinants of Base Editing Outcomes from Target Library Analysis and Machine Learning. Arbab, Mandana; Shen, Max W; Mok, Beverly; Wilson, Christopher; Matuszek, ?aneta; Cassa, Christopher A; Liu, David R Cell. 32533916 2020 Jul 23;
CHANGE-seq reveals genetic and epigenetic effects on CRISPR-Cas9 genome-wide activity. Lazzarotto, Cicera R; Malinin, Nikolay L; Li, Yichao; Zhang, Ruochi; Yang, Yang; Lee, GaHyun; Cowley, Eleanor; He, Yanghua; Lan, Xin; Jividen, Kasey; Katta, Varun; Kolmakova, Natalia G; Petersen, Christopher T; Qi, Qian; Strelcov, Evgheni; Maragh, Samanth Nature biotechnology. 32541958 2020 Jun 15;
Induced pluripotent stem cells provide mega insights into kidney disease. Luciani, Alessandro; Freedman, Benjamin S Kidney international 32571490 2020 07
Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors. Anzalone, Andrew V; Koblan, Luke W; Liu, David R Nature biotechnology. 32572269 2020 Jun 22;
Polymeric vehicles for nucleic acid delivery. Piotrowski-Daspit, Alexandra S; Kauffman, Amy C; Bracaglia, Laura G; Saltzman, W Mark Advanced drug delivery reviews. 32585159 2020 Jun 23;
Programmable m6A modification of cellular RNAs with a Cas13-directed methyltransferase. Wilson, Christopher; Chen, Peter J; Miao, Zhuang; Liu, David R Nature biotechnology. 32601430 2020 Jun 29;
The delivery challenge: fulfilling the promise of therapeutic genome editing. van Haasteren, Joost; Li, Jie; Scheideler, Olivia J; Murthy, Niren; Schaffer, David V Nature biotechnology. 32601435 2020 07;
Structural characterization of a novel human adeno-associated virus capsid with neurotropic properties. Hsu, Hung-Lun; Brown, Alexander; Loveland, Anna B; Lotun, Anoushka; Xu, Meiyu; Luo, Li; Xu, Guangchao; Li, Jia; Ren, Lingzhi; Su, Qin; Gessler, Dominic J; Wei, Yuquan; Tai, Phillip W L; Korostelev, Andrei A; Gao, Guangping Nature communications. 32606306 2020 Jun 30;
A bacterial cytidine deaminase toxin enables CRISPR-free mitochondrial base editing. Mok, Beverly Y; de Moraes, Marcos H; Zeng, Jun; Bosch, Dustin E; Kotrys, Anna V; Raguram, Aditya; Hsu, FoSheng; Radey, Matthew C; Peterson, S Brook; Mootha, Vamsi K; Mougous, Joseph D; Liu, David R Nature. 32641830 2020 07;
A scoutRNA Is Required for Some Type V CRISPR-Cas Systems. Harrington, Lucas B; Ma, Enbo; Chen, Janice S; Witte, Isaac P; Gertz, Dov; Paez-Espino, David; Al-Shayeb, Basem; Kyrpides, Nikos C; Burstein, David; Banfield, Jillian F; Doudna, Jennifer A Molecular cell. 32645367 2020 08 06;
Profiling APOL1 Nephropathy Risk Variants in Genome-Edited Kidney Organoids with Single-Cell Transcriptomics. Liu, Esther; Radmanesh, Behram; Chung, Byungha H; Donnan, Michael D; Yi, Dan; Dadi, Amal; Smith, Kelly D; Himmelfarb, Jonathan; Li, Mingyao; Freedman, Benjamin S; Lin, Jennie Kidney360. 32656538 2020 Mar;
Imidazole-Based Synthetic Lipidoids for In Vivo mRNA Delivery into Primary T Lymphocytes. Zhao, Xuewei; Chen, Jinjin; Qiu, Min; Li, Yamin; Glass, Zachary; Xu, Qiaobing Angewandte Chemie (International ed. in English) 32662132 2020 11 02
Advances in Alpha-1 Antitrypsin Gene Therapy. Lorincz, Reka; Curiel, David T American journal of respiratory cell and molecular biology. 32668173 2020 Nov;
Coevolution of Adeno-associated Virus Capsid Antigenicity and Tropism through a Structure-Guided Approach. Havlik, L Patrick; Simon, Katherine E; Smith, J Kennon; Klinc, Kelli A; Tse, Longping V; Oh, Daniel K; Fanous, Marco M; Meganck, Rita M; Mietzsch, Mario; Kleinschmidt, Jürgen; Agbandje-McKenna, Mavis; Asokan, Aravind Journal of virology. 32669336 2020 Sep 15;
CRISPR-Cas? from huge phages is a hypercompact genome editor. Pausch, Patrick; Al-Shayeb, Basem; Bisom-Rapp, Ezra; Tsuchida, Connor A; Li, Zheng; Cress, Brady F; Knott, Gavin J; Jacobsen, Steven E; Banfield, Jillian F; Doudna, Jennifer A Science (New York, N.Y.). 32675376 2020 07 17;
The dawn of non-human primate models for neurodevelopmental disorders. Aida, Tomomi; Feng, Guoping Current opinion in genetics & development 32693220 2020 12
Advances in microphysiological blood-brain barrier (BBB) models towards drug delivery. Lee, Caleb S; Leong, Kam W Current opinion in biotechnology. 32711361 2020 Dec;
DNA capture by a CRISPR-Cas9-guided adenine base editor. Lapinaite, Audrone; Knott, Gavin J; Palumbo, Cody M; Lin-Shiao, Enrique; Richter, Michelle F; Zhao, Kevin T; Beal, Peter A; Liu, David R; Doudna, Jennifer A Science (New York, N.Y.). 32732424 2020 07 31;
Epigenetic transcriptional reprogramming by WT1 mediates a repair response during podocyte injury. Ettou, Sandrine; Jung, Youngsook L; Miyoshi, Tomoya; Jain, Dhawal; Hiratsuka, Ken; Schumacher, Valerie; Taglienti, Mary E; Morizane, Ryuji; Park, Peter J; Kreidberg, Jordan A Science advances. 32754639 2020 Jul;
NDAT Targets PI3K-Mediated PD-L1 Upregulation to Reduce Proliferation in Gefitinib-Resistant Colorectal Cancer. Huang, Tung-Yung; Chang, Tung-Cheng; Chin, Yu-Tang; Pan, Yi-Shin; Chang, Wong-Jin; Liu, Feng-Cheng; Hastuti, Ema Dwi; Chiu, Shih-Jiuan; Wang, Shwu-Huey; Changou, Chun A; Li, Zi-Lin; Chen, Yi-Ru; Chu, Hung-Ru; Shih, Ya-Jung; Cheng, R Holland; Wu, Alexander Cells. 32756527 2020 08 03;
Treating Cystic Fibrosis with mRNA and CRISPR. Da Silva Sanchez, Alejandro; Paunovska, Kalina; Cristian, Ana; Dahlman, James E Human gene therapy 32799680 2020 09
New Directions in Pulmonary Gene Therapy. Vu, Amber; McCray , Jr, Paul B Human gene therapy 32814451 2020 09
mRNA Delivery Using Bioreducible Lipidoid Nanoparticles Facilitates Neural Differentiation of Human Mesenchymal Stem Cells. Zhao, Xuewei; Glass, Zachary; Chen, Jinjin; Yang, Liu; Kaplan, David L; Xu, Qiaobing Advanced healthcare materials 32815325 2021 02
The Golgi Calcium ATPase Pump Plays an Essential Role in Adeno-associated Virus Trafficking and Transduction. Madigan, Victoria J; Berry, Garrett E; Tyson, Tyne O; Nardone-White, Dasean; Ark, Jonathan; Elmore, Zachary C; Murlidharan, Giridhar; Vincent, Heather A; Asokan, Aravind Journal of virology. 32817219 2020 Oct 14;
Opportunities and limitations of genetically modified nonhuman primate models for neuroscience research. Feng, Guoping; Jensen, Frances E; Greely, Henry T; Okano, Hideyuki; Treue, Stefan; Roberts, Angela C; Fox, James G; Caddick, Sarah; Poo, Mu-Ming; Newsome, William T; Morrison, John H Proceedings of the National Academy of Sciences of the United States of America. 32817435 2020 09 29;
Single C-to-T substitution using engineered APOBEC3G-nCas9 base editors with minimum genome- and transcriptome-wide off-target effects. Lee, Sangsin; Ding, Ning; Sun, Yidi; Yuan, Tanglong; Li, Jing; Yuan, Qichen; Liu, Lizhong; Yang, Jie; Wang, Qian; Kolomeisky, Anatoly B; Hilton, Isaac B; Zuo, Erwei; Gao, Xue Science advances 32832622 2020 07
Neurotransmitter-derived lipidoids (NT-lipidoids) for enhanced brain delivery through intravenous injection. Ma, Feihe; Yang, Liu; Sun, Zhuorui; Chen, Jinjin; Rui, Xuehui; Glass, Zachary; Xu, Qiaobing Science advances 32832671 2020 07
Frame-Hydrogel Methodology for Engineering Highly Functional Cardiac Tissue Constructs. Helfer, Abbigail; Bursac, Nenad Methods in molecular biology (Clifton, N.J.). 32857373 2021;
Synthetic immunomodulation with a CRISPR super-repressor in vivo. Moghadam, Farzaneh; LeGraw, Ryan; Velazquez, Jeremy J; Yeo, Nan Cher; Xu, Chenxi; Park, Jin; Chavez, Alejandro; Ebrahimkhani, Mo R; Kiani, Samira Nature cell biology. 32884147 2020 Sep;
Adenoviral vectors for in vivo delivery of CRISPR-Cas gene editors. Boucher, Paul; Cui, Xiaoxia; Curiel, David T Journal of controlled release : official journal of the Controlled Release Society 32891680 2020 11 10
Rapid, precise quantification of large DNA excisions and inversions by ddPCR. Watry, Hannah L; Feliciano, Carissa M; Gjoni, Ketrin; Takahashi, Gou; Miyaoka, Yuichiro; Conklin, Bruce R; Judge, Luke M Scientific reports. 32913194 2020 09 10;
SARS-CoV-2 infection of human iPSC-derived cardiac cells predicts novel cytopathic features in hearts of COVID-19 patients. Pérez-Bermejo, Juan A; Kang, Serah; Rockwood, Sarah J; Simoneau, Camille R; Joy, David A; Ramadoss, Gokul N; Silva, Ana C; Flanigan, Will R; Li, Huihui; Nakamura, Ken; Whitman, Jeffrey D; Ott, Melanie; Conklin, Bruce R; McDevitt, Todd C bioRxiv : the preprint server for biology. 32935097 2020 Sep 12;
Rescuing AAV gene transfer from neutralizing antibodies with an IgG-degrading enzyme. Elmore, Zachary C; Oh, Daniel K; Simon, Katherine E; Fanous, Marco M; Asokan, Aravind JCI insight 32941184 2020 09 17
Serum Concentrations of Per- and Polyfluoroalkyl Substances and Risk of Renal Cell Carcinoma. Shearer, Joseph J; Callahan, Catherine L; Calafat, Antonia M; Huang, Wen-Yi; Jones, Rena R; Sabbisetti, Venkata S; Freedman, Neal D; Sampson, Joshua N; Silverman, Debra T; Purdue, Mark P; Hofmann, Jonathan N Journal of the National Cancer Institute 32944748 2021 May 04
Direct Functional Protein Delivery with a Peptide into Neonatal and Adult Mammalian Inner Ear In Vivo. Zhang, Kun; Cheng, Xiaoting; Zhao, Liping; Huang, Mingqian; Tao, Yong; Zhang, Hongbo; Rosenholm, Jessica M; Zhuang, Min; Chen, Zheng-Yi; Chen, Bing; Shu, Yilai Molecular therapy. Methods & clinical development. 32953927 2020 Sep 11;
Methods Matter: Standard Production Platforms for Recombinant AAV Produce Chemically and Functionally Distinct Vectors. Rumachik, Neil G; Malaker, Stacy A; Poweleit, Nicole; Maynard, Lucy H; Adams, Christopher M; Leib, Ryan D; Cirolia, Giana; Thomas, Dennis; Stamnes, Susan; Holt, Kathleen; Sinn, Patrick; May, Andrew P; Paulk, Nicole K Molecular therapy. Methods & clinical development. 32995354 2020 Sep 11;
COVID-19 CG: Tracking SARS-CoV-2 mutations by locations and dates of interest. Chen, Albert Tian; Altschuler, Kevin; Zhan, Shing Hei; Chan, Yujia Alina; Deverman, Benjamin E bioRxiv : the preprint server for biology. 32995794 2020 Sep 28;
Development of a Cardiac Sarcomere Functional Genomics Platform to Enable Scalable Interrogation of Human TNNT2 Variants. Pettinato, Anthony M; Ladha, Feria A; Mellert, David J; Legere, Nicholas; Cohn, Rachel; Romano, Robert; Thakar, Ketan; Chen, Yu-Sheng; Hinson, J Travis Circulation. 33025817 2020 Oct 07;
3D kidney organoids for bench-to-bedside translation. Gupta, Navin; Dilmen, Emre; Morizane, Ryuji Journal of molecular medicine (Berlin, Germany). 33034708 2020 Oct 09;
A Versatile Nonviral Delivery System for Multiplex Gene-Editing in the Liver. Gong, Jing; Wang, Hong-Xia; Lao, Yeh-Hsing; Hu, Hanze; Vatan, Naazanene; Guo, Jonathan; Ho, Tzu-Chieh; Huang, Dantong; Li, Mingqiang; Shao, Dan; Leong, Kam W Advanced materials (Deerfield Beach, Fla.) 33053221 2020 Nov
Reactions to the National Academies/Royal Society Report on Heritable Human Genome Editing. Angrist, Misha; Barrangou, Rodolphe; Baylis, Françoise; Brokowski, Carolyn; Burgio, Gaetan; Caplan, Arthur; Chapman, Carolyn Riley; Church, George M; Cook-Deegan, Robert; Cwik, Bryan; Doudna, Jennifer A; Evans, John H; Greely, Henry T; Hercher, Laura; Hurlbut, J Benjamin; Hynes, Richard O; Ishii, Tetsuya; Kiani, Samira; Lee, LaTasha Hoskins; Levrier, Guillaume; Liu, David R; Lunshof, Jeantine E; Macintosh, Kerry Lynn; Mathews, Debra J H; Meslin, Eric M; Mills, Peter H R; Montoliu, Lluis; Musunuru, Kiran; Nicol, Dianne; O'Neill, Helen; Qiu, Renzong; Ranisch, Robert; Sherkow, Jacob S; Soni, Sheetal; Terry, Sharon; Topol, Eric; Williamson, Robert; Zhang, Feng; Davies, Kevin The CRISPR journal 33095048 2020 10
In Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular Dystrophy. Kwon, Jennifer B; Ettyreddy, Adarsh R; Vankara, Ashish; Bohning, Joel D; Devlin, Garth; Hauschka, Stephen D; Asokan, Aravind; Gersbach, Charles A Molecular therapy. Methods & clinical development. 33145368 2020 Dec 11;
Poison Exon Splicing Regulates a Coordinated Network of SR Protein Expression during Differentiation and Tumorigenesis. Leclair, Nathan K; Brugiolo, Mattia; Urbanski, Laura; Lawson, Shane C; Thakar, Ketan; Yurieva, Marina; George, Joshy; Hinson, John Travis; Cheng, Albert; Graveley, Brenton R; Anczuków, Olga Molecular cell. 33176162 2020 11 19;
Redirecting Vesicular Transport to Improve Nonviral Delivery of Molecular Cargo. Mao, Mao; Chang, Chun-Chi; Pickar-Oliver, Adrian; Cervia, Lisa D; Wang, Liangli; Ji, Jing; Liton, Paloma B; Gersbach, Charles A; Yuan, Fan Advanced biosystems 33179869 2020 08
Delayed urea differential enhancement CEST (dudeCEST)-MRI with T1 correction for monitoring renal urea handling. Shin, Soo Hyun; Wendland, Michael F; Vandsburger, Moriel H Magnetic resonance in medicine. 33180343 2020 Nov 12;
The once and future gene therapy. Bulaklak, Karen; Gersbach, Charles A Nature communications. 33199717 2020 11 16;
Phage-assisted continuous and non-continuous evolution. Miller, Shannon M; Wang, Tina; Liu, David R Nature protocols. 33199872 2020 Dec;
Master Regulators and Cofactors of Human Neuronal Cell Fate Specification Identified by CRISPR Gene Activation Screens. Black, Joshua B; McCutcheon, Sean R; Dube, Shataakshi; Barrera, Alejandro; Klann, Tyler S; Rice, Grayson A; Adkar, Shaunak S; Soderling, Scott H; Reddy, Timothy E; Gersbach, Charles A Cell reports. 33264623 2020 Dec 01;
Tools for experimental and computational analyses of off-target editing by programmable nucleases. Bao, X Robert; Pan, Yidan; Lee, Ciaran M; Davis, Timothy H; Bao, Gang Nature protocols. 33288953 2020 Dec 07;
Gene Regulatory Network Analysis and Engineering Directs Development and Vascularization of Multilineage Human Liver Organoids. Velazquez, Jeremy J; LeGraw, Ryan; Moghadam, Farzaneh; Tan, Yuqi; Kilbourne, Jacquelyn; Maggiore, Joseph C; Hislop, Joshua; Liu, Silvia; Cats, Davy; Chuva de Sousa Lopes, Susana M; Plaisier, Christopher; Cahan, Patrick; Kiani, Samira; Ebrahimkhani, Mo R Cell systems 33290741 2021 Jan 20
Targeting de novo purine synthesis pathway via ADSL depletion impairs liver cancer growth by perturbing mitochondrial function. Jiang, Tingting; Sánchez-Rivera, Francisco J; Soto-Feliciano, Yadira M; Yang, Qiyuan; Song, Chun-Qing; Bhuatkar, Arjun; Haynes, Cole M; Hemann, Michael T; Xue, Wen Hepatology (Baltimore, Md.) 33336367 2020 Dec 17
Overcoming innate immune barriers that impede AAV gene therapy vectors. Muhuri, Manish; Maeda, Yukiko; Ma, Hong; Ram, Sanjay; Fitzgerald, Katherine A; Tai, Phillip Wl; Gao, Guangping The Journal of clinical investigation 33393506 2021 Jan 04
In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice. Koblan, Luke W; Erdos, Michael R; Wilson, Christopher; Cabral, Wayne A; Levy, Jonathan M; Xiong, Zheng-Mei; Tavarez, Urraca L; Davison, Lindsay M; Gete, Yantenew G; Mao, Xiaojing; Newby, Gregory A; Doherty, Sean P; Narisu, Narisu; Sheng, Quanhu; Krilow, Chad; Lin, Charles Y; Gordon, Leslie B; Cao, Kan; Collins, Francis S; Brown, Jonathan D; Liu, David R Nature 33408413 2021 Jan
CRISPR/Cas9 gene editing for curing sickle cell disease. Park, So Hyun; Bao, Gang Transfusion and apheresis science : official journal of the World Apheresis Association : official journal of the European Society for Haemapheresis 33455878 2021 Jan 10
Precision genome editing using cytosine and adenine base editors in mammalian cells. Huang, Tony P; Newby, Gregory A; Liu, David R Nature protocols 33462442 2021 Feb
Determining of canine position by multiple facial landmarks to achieve natural esthetics in complete denture treatment. Srimaneekarn, Natchalee; Arayapisit, Tawepong; Pooktuantong, Ornnicha; Cheng, Holland R; Soonsawad, Pan The Journal of prosthetic dentistry 33468316 2021 Jan 16
Nanoparticles for delivery of agents to fetal lungs. Ullrich, Sarah J; Freedman-Weiss, Mollie; Ahle, Samantha; Mandl, Hanna K; Piotrowski-Daspit, Alexandra S; Roberts, Katherine; Yung, Nicholas; Maassel, Nathan; Bauer-Pisani, Tory; Ricciardi, Adele S; Egan, Marie E; Glazer, Peter M; Saltzman, W Mark; Stitelman, David H Acta biomaterialia 33484911 2021 Jan 21
Exercise mimetics and JAK inhibition attenuate IFN-γ-induced wasting in engineered human skeletal muscle. Chen, Zhaowei; Li, Binjie; Zhan, Ren-Zhi; Rao, Lingjun; Bursac, Nenad Science advances 33523949 2021 Jan
Gene-based therapies for neurodegenerative diseases. Sun, Jichao; Roy, Subhojit Nature neuroscience 33526943 2021 03
Viral vector platforms within the gene therapy landscape. Bulcha, Jote T; Wang, Yi; Ma, Hong; Tai, Phillip W L; Gao, Guangping Signal transduction and targeted therapy 33558455 2021 Feb 08
Soluble CD40L activates soluble and cell-surface integrins αvβ3, α5β1 and α4β1 by binding to the allosteric ligand-binding site (site 2). Takada, Yoko K; Shimoda, Michiko; Maverakis, Emanual; Felding, Brunie H; Cheng, R Holland; Takada, Yoshikazu The Journal of biological chemistry 33571526 2021 Feb 08
PrimeDesign software for rapid and simplified design of prime editing guide RNAs. Hsu, Jonathan Y; Grünewald, Julian; Szalay, Regan; Shih, Justine; Anzalone, Andrew V; Lam, Kin Chung; Shen, Max W; Petri, Karl; Liu, David R; Keith Joung, J; Pinello, Luca Nature communications 33589617 2021 02 15
Massively parallel assessment of human variants with base editor screens. Hanna, Ruth E; Hegde, Mudra; Fagre, Christian R; DeWeirdt, Peter C; Sangree, Annabel K; Szegletes, Zsofia; Griffith, Audrey; Feeley, Marissa N; Sanson, Kendall R; Baidi, Yossef; Koblan, Luke W; Liu, David R; Neal, James T; Doench, John G Cell 33606977 2021 02 18
Engineering highly efficient backsplicing and translation of synthetic circRNAs. Meganck, Rita M; Liu, Jiacheng; Hale, Andrew E; Simon, Katherine E; Fanous, Marco M; Vincent, Heather A; Wilusz, Jeremy E; Moorman, Nathaniel J; Marzluff, William F; Asokan, Aravind Molecular therapy. Nucleic acids 33614232 2021 Mar 05
COVID-19 CG enables SARS-CoV-2 mutation and lineage tracking by locations and dates of interest. Chen, Albert Tian; Altschuler, Kevin; Zhan, Shing Hei; Chan, Yujia Alina; Deverman, Benjamin E eLife 33620031 2021 02 23
Lipid nanoparticle-mediated codelivery of Cas9 mRNA and single-guide RNA achieves liver-specific in vivo genome editing of Angptl3. Qiu, Min; Glass, Zachary; Chen, Jinjin; Haas, Mary; Jin, Xin; Zhao, Xuewei; Rui, Xuehui; Ye, Zhongfeng; Li, Yamin; Zhang, Feng; Xu, Qiaobing Proceedings of the National Academy of Sciences of the United States of America 33649229 2021 Mar 09
Therapeutic RNA Delivery for COVID and Other Diseases. Dobrowolski, Curtis; Paunovska, Kalina; Hatit, Marine Z C; Lokugamage, Melissa P; Dahlman, James E Advanced healthcare materials 33661555 2021 Mar 04
A CRISPR-Cas9-integrase complex generates precise DNA fragments for genome integration. Jakhanwal, Shrutee; Cress, Brady F; Maguin, Pascal; Lobba, Marco J; Marraffini, Luciano A; Doudna, Jennifer A Nucleic acids research 33693715 2021 04 06
Loss of sarcomeric proteins via upregulation of JAK/STAT signaling underlies interferon-γ-induced contractile deficit in engineered human myocardium. Zhan, Ren-Zhi; Rao, Lingjun; Chen, Zhaowei; Strash, Nicholas; Bursac, Nenad Acta biomaterialia 33705988 2021 05
AAV-Mediated Gene Therapy for Glycosphingolipid Biosynthesis Deficiencies. Yang, Huiya; Brown Jr, Robert H; Wang, Dan; Strauss, Kevin A; Gao, Guangping Trends in molecular medicine 33714697 2021 06
Tissue-Engineered Skeletal Muscle Models to Study Muscle Function, Plasticity, and Disease. Khodabukus, Alastair Frontiers in physiology 33716768 2021
Multicellular Systems to Translate Somatic Cell Genome Editors to Humans. Hernandez-Gordillo, Victor; Casolaro, Thomas Caleb; Ebrahimkhani, Mo R; Kiani, Samira Current opinion in biomedical engineering 33718690 2020 Dec
Prime editing in mice reveals the essentiality of a single base in driving tissue-specific gene expression. Gao, Pan; Lyu, Qing; Ghanam, Amr R; Lazzarotto, Cicera R; Newby, Gregory A; Zhang, Wei; Choi, Mihyun; Slivano, Orazio J; Holden, Kevin; Walker 2nd, John A; Kadina, Anastasia P; Munroe, Rob J; Abratte, Christian M; Schimenti, John C; Liu, David R; Tsai, Shengdar Q; Long, Xiaochun; Miano, Joseph M Genome biology 33722289 2021 03 16
SARS-CoV-2 infection of human iPSC-derived cardiac cells reflects cytopathic features in hearts of patients with COVID-19. Perez-Bermejo, Juan A; Kang, Serah; Rockwood, Sarah J; Simoneau, Camille R; Joy, David A; Silva, Ana C; Ramadoss, Gokul N; Flanigan, Will R; Fozouni, Parinaz; Li, Huihui; Chen, Pei-Yi; Nakamura, Ken; Whitman, Jeffrey D; Hanson, Paul J; McManus, Bruce M; Ott, Melanie; Conklin, Bruce R; McDevitt, Todd C Science translational medicine 33723017 2021 04 21
Nanoparticles containing constrained phospholipids deliver mRNA to liver immune cells in vivo without targeting ligands. Gan, Zubao; Lokugamage, Melissa P; Hatit, Marine Z C; Loughrey, David; Paunovska, Kalina; Sato, Manaka; Cristian, Ana; Dahlman, James E Bioengineering & translational medicine 33758781 2020 Sep
Increased CFTR expression and function from an optimized lentiviral vector for cystic fibrosis gene therapy. Marquez Loza, Laura I; Cooney, Ashley L; Dong, Qian; Randak, Christoph O; Rivella, Stefano; Sinn, Patrick L; McCray Jr, Paul B Molecular therapy. Methods & clinical development 33768133 2021 Jun 11
Large-scale molecular epidemiological analysis of AAV in a cancer patient population. Qin, Wanru; Xu, Guangchao; Tai, Phillip W L; Wang, Chunmei; Luo, Li; Li, Chengjian; Hu, Xun; Xue, Jianxin; Lu, You; Zhou, Qiao; Wei, Qiang; Wen, Tianfu; Hu, Jiankun; Xiao, Yuanyuan; Yang, Li; Li, Weimin; Flotte, Terence R; Wei, Yuquan; Gao, Guangping Oncogene 33782545 2021 Apr
PEGylation of poly(amine-co-ester) polyplexes for tunable gene delivery. Grun, Molly K; Suberi, Alexandra; Shin, Kwangsoo; Lee, Teresa; Gomerdinger, Victoria; Moscato, Zoe M; Piotrowski-Daspit, Alexandra S; Saltzman, W Mark Biomaterials 33813260 2021 May
Examining the evidence for extracellular RNA function in mammals. Gruner, Hannah N; McManus, Michael T Nature reviews. Genetics 33824487 2021 07
The NIH Somatic Cell Genome Editing program. Saha, Krishanu; Sontheimer, Erik J; Brooks, P J; Dwinell, Melinda R; Gersbach, Charles A; Liu, David R; Murray, Stephen A; Tsai, Shengdar Q; Wilson, Ross C; Anderson, Daniel G; Asokan, Aravind; Banfield, Jillian F; Bankiewicz, Krystof S; Bao, Gang; Bulte, Jeff W M; Bursac, Nenad; Campbell, Jarryd M; Carlson, Daniel F; Chaikof, Elliot L; Chen, Zheng-Yi; Cheng, R Holland; Clark, Karl J; Curiel, David T; Dahlman, James E; Deverman, Benjamin E; Dickinson, Mary E; Doudna, Jennifer A; Ekker, Stephen C; Emborg, Marina E; Feng, Guoping; Freedman, Benjamin S; Gamm, David M; Gao, Guangping; Ghiran, Ionita C; Glazer, Peter M; Gong, Shaoqin; Heaney, Jason D; Hennebold, Jon D; Hinson, John T; Khvorova, Anastasia; Kiani, Samira; Lagor, William R; Lam, Kit S; Leong, Kam W; Levine, Jon E; Lewis, Jennifer A; Lutz, Cathleen M; Ly, Danith H; Maragh, Samantha; McCray Jr, Paul B; McDevitt, Todd C; Mirochnitchenko, Oleg; Morizane, Ryuji; Murthy, Niren; Prather, Randall S; Ronald, John A; Roy, Subhojit; Roy, Sushmita; Sabbisetti, Venkata; Saltzman, W Mark; Santangelo, Philip J; Segal, David J; Shimoyama, Mary; Skala, Melissa C; Tarantal, Alice F; Tilton, John C; Truskey, George A; Vandsburger, Moriel; Watts, Jonathan K; Wells, Kevin D; Wolfe, Scot A; Xu, Qiaobing; Xue, Wen; Yi, Guohua; Zhou, Jiangbing; SCGE Consortium Nature 33828315 2021 Apr
Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice. Liu, Pengpeng; Liang, Shun-Qing; Zheng, Chunwei; Mintzer, Esther; Zhao, Yan G; Ponnienselvan, Karthikeyan; Mir, Aamir; Sontheimer, Erik J; Gao, Guangping; Flotte, Terence R; Wolfe, Scot A; Xue, Wen Nature communications 33837189 2021 04 09
VectorMOD: Method for Bottom-Up Proteomic Characterization of rAAV Capsid Post-Translational Modifications and Vector Impurities. Rumachik, Neil G; Malaker, Stacy A; Paulk, Nicole K Frontiers in immunology 33868302 2021
Integrating Biomaterials and Genome Editing Approaches to Advance Biomedical Science. Abdeen, Amr A; Cosgrove, Brian D; Gersbach, Charles A; Saha, Krishanu Annual review of biomedical engineering 33909475 2021 07 13
Sarcomere function activates a p53-dependent DNA damage response that promotes polyploidization and limits in vivo cell engraftment. Pettinato, Anthony M; Yoo, Dasom; VanOudenhove, Jennifer; Chen, Yu-Sheng; Cohn, Rachel; Ladha, Feria A; Yang, Xiulan; Thakar, Ketan; Romano, Robert; Legere, Nicolas; Meredith, Emily; Robson, Paul; Regnier, Michael; Cotney, Justin L; Murry, Charles E; Hins Cell reports 33951429 2021 May 04
Three-dimensional tissue-engineered human skeletal muscle model of Pompe disease. Wang, Jason; Zhou, Chris J; Khodabukus, Alastair; Tran, Sabrina; Han, Sang-Oh; Carlson, Aaron L; Madden, Lauran; Kishnani, Priya S; Koeberl, Dwight D; Bursac, Nenad Communications biology 33953320 2021 05 05
3D cell culture models: Drug pharmacokinetics, safety assessment, and regulatory consideration. Wang, Hongbing; Brown, Paul C; Chow, Edwin C Y; Ewart, Lorna; Ferguson, Stephen S; Fitzpatrick, Suzanne; Freedman, Benjamin S; Guo, Grace L; Hedrich, William; Heyward, Scott; Hickman, James; Isoherranen, Nina; Li, Albert P; Liu, Qi; Mumenthaler, Shannon M; Polli, James; Proctor, William R; Ribeiro, Alexandre; Wang, Jian-Ying; Wange, Ronald L; Huang, Shiew-Mei Clinical and translational science 33982436 2021 09
Novel Combinatorial MicroRNA-Binding Sites in AAV Vectors Synergistically Diminish Antigen Presentation and Transgene Immunity for Efficient and Stable Transduction. Muhuri, Manish; Zhan, Wei; Maeda, Yukiko; Li, Jia; Lotun, Anoushka; Chen, Jennifer; Sylvia, Katelyn; Dasgupta, Ishani; Arjomandnejad, Motahareh; Nixon, Thomas; Keeler, Allison M; Manokaran, Sangeetha; He, Ran; Su, Qin; Tai, Phillip W L; Gao, Guangping Frontiers in immunology 33995418 2021
Efficient embryonic homozygous gene conversion via RAD51-enhanced interhomolog repair. Wilde, Jonathan J; Aida, Tomomi; Del Rosario, Ricardo C H; Kaiser, Tobias; Qi, Peimin; Wienisch, Martin; Zhang, Qiangge; Colvin, Steven; Feng, Guoping Cell 34043941 2021 Jun 10
Vectored Immunotherapeutics for Infectious Diseases: Can rAAVs Be The Game Changers for Fighting Transmissible Pathogens? Zhan, Wei; Muhuri, Manish; Tai, Phillip W L; Gao, Guangping Frontiers in immunology 34046041 2021
Quantification of Cellular Densities and Antigenic Properties using Magnetic Levitation. Thompson, Lauren; Pinckney, Brandy; Lu, Shulin; Gregory, Mark; Tigges, John; Ghiran, Ionita Journal of visualized experiments : JoVE 34057455 2021 05 17
Electrophoretic mobility shift as a molecular beacon-based readout for miRNA detection. Oliveira-Jr, Getulio P; Barbosa, Raquel H; Thompson, Lauren; Pinckney, Brandy; Murphy-Thornley, Moriah; Lu, Shulin; Jones, Jennifer; Hansen, Clinton H; Tigges, John; Wong, Wesley P; Ghiran, Ionita C Biosensors & bioelectronics 34062334 2021 Oct 01
Generation and Genetic Correction of USH2A c.2299delG Mutation in Patient-Derived Induced Pluripotent Stem Cells. Liu, Xuezhong; Lillywhite, Justin; Zhu, Wenliang; Huang, Zaohua; Clark, Anna M; Gosstola, Nicholas; Maguire, Colin T; Dykxhoorn, Derek; Chen, Zheng-Yi; Yang, Jun Genes 34070435 2021 05 25
SARS-CoV-2 infection induces the activation of tissue factor-mediated coagulation via activation of acid sphingomyelinase. Wang, Jue; Pendurthi, Usha R; Yi, Guohua; Rao, L Vijaya Mohan Blood 34075401 2021 07 29
Targeted delivery of CRISPR-Cas9 and transgenes enables complex immune cell engineering. Hamilton, Jennifer R; Tsuchida, Connor A; Nguyen, David N; Shy, Brian R; McGarrigle, E Riley; Sandoval Espinoza, Cindy R; Carr, Daniel; Blaeschke, Franziska; Marson, Alexander; Doudna, Jennifer A Cell reports 34077734 2021 Jun 01
Escaping the endosome: assessing cellular trafficking mechanisms of non-viral vehicles. Xu, Emily; Saltzman, W Mark; Piotrowski-Daspit, Alexandra S Journal of controlled release : official journal of the Controlled Release Society 34077782 2021 07 10
Base editing of haematopoietic stem cells rescues sickle cell disease in mice. Newby, Gregory A; Yen, Jonathan S; Woodard, Kaitly J; Mayuranathan, Thiyagaraj; Lazzarotto, Cicera R; Li, Yichao; Sheppard-Tillman, Heather; Porter, Shaina N; Yao, Yu; Mayberry, Kalin; Everette, Kelcee A; Jang, Yoonjeong; Podracky, Christopher J; Thaman, Elizabeth; Lechauve, Christophe; Sharma, Akshay; Henderson, Jordana M; Richter, Michelle F; Zhao, Kevin T; Miller, Shannon M; Wang, Tina; Koblan, Luke W; McCaffrey, Anton P; Tisdale, John F; Kalfa, Theodosia A; Pruett-Miller, Shondra M; Tsai, Shengdar Q; Weiss, Mitchell J; Liu, David R Nature 34079130 2021 Jul
Mechanisms of angiogenic incompetence in Hutchinson-Gilford progeria via downregulation of endothelial NOS. Gete, Yantenew G; Koblan, Luke W; Mao, Xiaojing; Trappio, Mason; Mahadik, Bhushan; Fisher, John P; Liu, David R; Cao, Kan Aging cell 34086398 2021 07
Targeting the Apoa1 locus for liver-directed gene therapy. De Giorgi, Marco; Li, Ang; Hurley, Ayrea; Barzi, Mercedes; Doerfler, Alexandria M; Cherayil, Nikitha A; Smith, Harrison E; Brown, Jonathan D; Lin, Charles Y; Bissig, Karl-Dimiter; Bao, Gang; Lagor, William R Molecular therapy. Methods & clinical development 34141821 2021 Jun 11
Magnetic Forces Enable Control of Biological Processes In Vivo. Bao, Gang Journal of applied mechanics 34168385 2021 Mar 01
In vivo targeted delivery of nucleic acids and CRISPR genome editors enabled by GSH-responsive silica nanoparticles. Wang, Yuyuan; Shahi, Pawan K; Wang, Xiuxiu; Xie, Ruosen; Zhao, Yi; Wu, Min; Roge, Seth; Pattnaik, Bikash R; Gong, Shaoqin Journal of controlled release : official journal of the Controlled Release Society 34174352 2021 Jun 23
Prediction and validation of hematopoietic stem and progenitor cell off-target editing in transplanted rhesus macaques. AlJanahi, Aisha A; Lazzarotto, Cicera R; Chen, Shirley; Shin, Tae-Hoon; Cordes, Stefan; Fan, Xing; Jabara, Isabel; Zhou, Yifan; Young, David J; Lee, Byung-Chul; Yu, Kyung-Rok; Li, Yuesheng; Toms, Bradley; Tunc, Ilker; Hong, So Gun; Truitt, Lauren L; Klermund, Julia; Andrieux, Geoffroy; Kim, Miriam Y; Cathomen, Toni; Gill, Saar; Tsai, Shengdar Q; Dunbar, Cynthia E Molecular therapy : the journal of the American Society of Gene Therapy 34174439 2022 01 05
Controlling CRISPR with small molecule regulation for somatic cell genome editing. Khajanchi, Namita; Saha, Krishanu Molecular therapy : the journal of the American Society of Gene Therapy 34174442 2021 Jun 24
Gene editing in a Myo6 semi-dominant mouse model rescues auditory function. Xue, Yuanyuan; Hu, Xinde; Wang, Daqi; Li, Di; Li, Yige; Wang, Fang; Huang, Mingqian; Gu, Xi; Xu, Zhijiao; Zhou, Jinan; Wang, Jinghan; Chai, Renjie; Shen, Jun; Chen, Zheng-Yi; Li, Geng-Lin; Yang, Hui; Li, Huawei; Zuo, Erwei; Shu, Yilai Molecular therapy : the journal of the American Society of Gene Therapy 34174443 2021 Jun 24
Efficient C•G-to-G•C base editors developed using CRISPRi screens, target-library analysis, and machine learning. Koblan, Luke W; Arbab, Mandana; Shen, Max W; Hussmann, Jeffrey A; Anzalone, Andrew V; Doman, Jordan L; Newby, Gregory A; Yang, Dian; Mok, Beverly; Replogle, Joseph M; Xu, Albert; Sisley, Tyler A; Weissman, Jonathan S; Adamson, Britt; Liu, David R Nature biotechnology 34183861 2021 Jun 28
Mucopolysaccharidoses type I gene therapy. Hurt, Sarah C; Dickson, Patricia I; Curiel, David T Journal of inherited metabolic disease 34189746 2021 Jun 29
rAAV-based and intraprostatically delivered miR-34a therapeutics for efficient inhibition of prostate cancer progression. Ai, Jianzhong; Li, Jia; Su, Qin; Ma, Hong; He, Ran; Wei, Qiang; Li, Hong; Gao, Guangping Gene therapy 34226687 2021 Jul 06
Receptor Switching in Newly Evolved Adeno-associated Viruses. Havlik, L Patrick; Das, Anshuman; Mietzsch, Mario; Oh, Daniel K; Ark, Jonathan; McKenna, Robert; Agbandje-McKenna, Mavis; Asokan, Aravind Journal of virology 34232726 2021 09 09
Recent innovations in fluorescence lifetime imaging microscopy for biology and medicine. Datta, Rupsa; Gillette, Amani; Stefely, Matthew; Skala, Melissa C Journal of biomedical optics 34247457 2021 07
External stimuli-responsive nanoparticles for spatially and temporally controlled delivery of CRISPR-Cas genome editors. Xie, Ruosen; Wang, Yuyuan; Gong, Shaoqin Biomaterials science 34286726 2021 Sep 14
Vector Strategies to Actualize B Cell-Based Gene Therapies. Jeske, Amanda M; Boucher, Paul; Curiel, David T; Voss, James E Journal of immunology (Baltimore, Md. : 1950) 34321286 2021 08 01
Label-free imaging for quality control of cardiomyocyte differentiation. Qian, Tongcheng; Heaster, Tiffany M; Houghtaling, Angela R; Sun, Kexin; Samimi, Kayvan; Skala, Melissa C Nature communications 34321477 2021 07 28
Transgenic mice for in vivo epigenome editing with CRISPR-based systems. Gemberling, Matthew P; Siklenka, Keith; Rodriguez, Erica; Tonn-Eisinger, Katherine R; Barrera, Alejandro; Liu, Fang; Kantor, Ariel; Li, Liqing; Cigliola, Valentina; Hazlett, Mariah F; Williams, Courtney A; Bartelt, Luke C; Madigan, Victoria J; Bodle, Josephine C; Daniels, Heather; Rouse, Douglas C; Hilton, Isaac B; Asokan, Aravind; Ciofani, Maria; Poss, Kenneth D; Reddy, Timothy E; West, Anne E; Gersbach, Charles A Nature methods 34341582 2021 08
Actinin BioID reveals sarcomere crosstalk with oxidative metabolism through interactions with IGF2BP2. Ladha, Feria A; Thakar, Ketan; Pettinato, Anthony M; Legere, Nicholas; Ghahremani, Shahnaz; Cohn, Rachel; Romano, Robert; Meredith, Emily; Chen, Yu-Sheng; Hinson, J Travis Cell reports 34380038 2021 Aug 10
DNA interference states of the hypercompact CRISPR-CasΦ effector. Pausch, Patrick; Soczek, Katarzyna M; Herbst, Dominik A; Tsuchida, Connor A; Al-Shayeb, Basem; Banfield, Jillian F; Nogales, Eva; Doudna, Jennifer A Nature structural & molecular biology 34381246 2021 08
A Versatile and Robust Platform for the Scalable Manufacture of Biomimetic Nanovaccines. Hu, Hanze; Yang, Chao; Zhang, Fan; Li, Mingqiang; Tu, Zhaoxu; Mu, Lizhong; Dawulieti, Jianati; Lao, Yeh-Hsing; Xiao, Zixuan; Yan, Huize; Sun, Wen; Shao, Dan; Leong, Kam W Advanced science (Weinheim, Baden-Wurttemberg, Germany) 34386315 2021 08
Bioengineered optogenetic model of human neuromuscular junction. Vila, Olaia F; Chavez, Miguel; Ma, Stephen P; Yeager, Keith; Zholudeva, Lyandysha V; Colón-Mercado, Jennifer M; Qu, Yihuai; Nash, Trevor R; Lai, Carmen; Feliciano, Carissa M; Carter, Matthew; Kamm, Roger D; Judge, Luke M; Conklin, Bruce R; Ward, Michael E; McDevitt, Todd C; Vunjak-Novakovic, Gordana Biomaterials 34403849 2021 09
Easy-Prime: a machine learning-based prime editor design tool. Li, Yichao; Chen, Jingjing; Tsai, Shengdar Q; Cheng, Yong Genome biology 34412673 2021 08 19
Synthetic Biology Approaches for Engineering Next-Generation Adenoviral Gene Therapies. Collins, Logan Thrasher; Curiel, David T ACS nano 34415739 2021 09 28
Flash Technology-Based Self-Assembly in Nanoformulation: From Fabrication to Biomedical Applications. Hu, Hanze; Yang, Chao; Li, Mingqiang; Shao, Dan; Mao, Hai-Quan; Leong, Kam W Materials today (Kidlington, England) 34421329 2021 Jan-Feb
Cortical overgrowth in a preclinical forebrain organoid model of CNTNAP2-associated autism spectrum disorder. de Jong, Job O; Llapashtica, Ceyda; Genestine, Matthieu; Strauss, Kevin; Provenzano, Frank; Sun, Yan; Zhu, Huixiang; Cortese, Giuseppe P; Brundu, Francesco; Brigatti, Karlla W; Corneo, Barbara; Migliori, Bianca; Tomer, Raju; Kushner, Steven A; Kellendonk, Christoph; Javitch, Jonathan A; Xu, Bin; Markx, Sander Nature communications 34471112 2021 09 01
Light: A Magical Tool for Controlled Drug Delivery. Tao, Yu; Chan, Hon Fai; Shi, Bingyang; Li, Mingqiang; Leong, Kam W Advanced functional materials 34483808 2020 Dec 01
Allele-Specific Gene Editing Rescues Pathology in a Human Model of Charcot-Marie-Tooth Disease Type 2E. Feliciano, Carissa M; Wu, Kenneth; Watry, Hannah L; Marley, Chiara B E; Ramadoss, Gokul N; Ghanim, Hana Y; Liu, Angela Z; Zholudeva, Lyandysha V; McDevitt, Todd C; Saporta, Mario A; Conklin, Bruce R; Judge, Luke M Frontiers in cell and developmental biology 34485306 2021
Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy. Pickar-Oliver, Adrian; Gough, Veronica; Bohning, Joel D; Liu, Siyan; Robinson-Hamm, Jacqueline N; Daniels, Heather; Majoros, William H; Devlin, Garth; Asokan, Aravind; Gersbach, Charles A Molecular therapy : the journal of the American Society of Gene Therapy 34509668 2021 Nov 03
In vivo somatic cell base editing and prime editing. Newby, Gregory A; Liu, David R Molecular therapy : the journal of the American Society of Gene Therapy 34509669 2021 Nov 03
Functional correction of CFTR mutations in human airway epithelial cells using adenine base editors. Krishnamurthy, Sateesh; Traore, Soumba; Cooney, Ashley L; Brommel, Christian M; Kulhankova, Katarina; Sinn, Patrick L; Newby, Gregory A; Liu, David R; McCray, Paul B Nucleic acids research 34520545 2021 Oct 11
In Vivo MRI Tracking of Tumor Vaccination and Antigen Presentation by Dendritic Cells. Bulte, Jeff W M; Shakeri-Zadeh, Ali Molecular imaging and biology 34581954 2021 Sep 28
Engineered pegRNAs improve prime editing efficiency. Nelson, James W; Randolph, Peyton B; Shen, Simon P; Everette, Kelcee A; Chen, Peter J; Anzalone, Andrew V; An, Meirui; Newby, Gregory A; Chen, Jonathan C; Hsu, Alvin; Liu, David R Nature biotechnology 34608327 2021 Oct 04
Optimization of lipid nanoparticles for the delivery of nebulized therapeutic mRNA to the lungs. Lokugamage, Melissa P; Vanover, Daryll; Beyersdorf, Jared; Hatit, Marine Z C; Rotolo, Laura; Echeverri, Elisa Schrader; Peck, Hannah E; Ni, Huanzhen; Yoon, Jeong-Kee; Kim, YongTae; Santangelo, Philip J; Dahlman, James E Nature biomedical engineering 34616046 2021 09
Kidney development to kidney organoids and back again. Gupta, Navin; Morizane, Ryuji Seminars in cell & developmental biology 34627669 2021 Oct 06
Surface Topography of Polyethylene Glycol Shell Nanoparticles Formed from Bottlebrush Block Copolymers Controls Interactions with Proteins and Cells. Grundler, Julian; Shin, Kwangsoo; Suh, Hee-Won; Zhong, Mingjiang; Saltzman, W Mark ACS nano 34633171 2021 10 26
Disulfide-compatible phage-assisted continuous evolution in the periplasmic space. Morrison, Mary S; Wang, Tina; Raguram, Aditya; Hemez, Colin; Liu, David R Nature communications 34645844 2021 10 13
Deletion and replacement of long genomic sequences using prime editing. Jiang, Tingting; Zhang, Xiao-Ou; Weng, Zhiping; Xue, Wen Nature biotechnology 34650270 2021 Oct 14
Enhanced prime editing systems by manipulating cellular determinants of editing outcomes. Chen, Peter J; Hussmann, Jeffrey A; Yan, Jun; Knipping, Friederike; Ravisankar, Purnima; Chen, Pin-Fang; Chen, Cidi; Nelson, James W; Newby, Gregory A; Sahin, Mustafa; Osborn, Mark J; Weissman, Jonathan S; Adamson, Britt; Liu, David R Cell 34653350 2021 Oct 28
SnRNA sequencing defines signaling by RBC-derived extracellular vesicles in the murine heart. Valkov, Nedyalka; Das, Avash; Tucker, Nathan R; Li, Guoping; Salvador, Ane M; Chaffin, Mark D; Pereira De Oliveira Junior, Getulio; Kur, Ivan; Gokulnath, Priyanka; Ziegler, Olivia; Yeri, Ashish; Lu, Shulin; Khamesra, Aushee; Xiao, Chunyang; Rodosthenous, Life science alliance 34663679 2021 Dec
5'-Modifications improve potency and efficacy of DNA donors for precision genome editing. Ghanta, Krishna S; Chen, Zexiang; Mir, Aamir; Dokshin, Gregoriy A; Krishnamurthy, Pranathi M; Yoon, Yeonsoo; Gallant, Judith; Xu, Ping; Zhang, Xiao-Ou; Ozturk, Ahmet Rasit; Shin, Masahiro; Idrizi, Feston; Liu, Pengpeng; Gneid, Hassan; Edraki, Alireza; Lawson, Nathan D; Rivera-Pérez, Jaime A; Sontheimer, Erik J; Watts, Jonathan K; Mello, Craig C eLife 34665130 2021 10 19
Developing Biodegradable Lipid Nanoparticles for Intracellular mRNA Delivery and Genome Editing. Qiu, Min; Li, Yamin; Bloomer, Hanan; Xu, Qiaobing Accounts of chemical research 34668716 2021 11 02
AAV5 delivery of CRISPR-Cas9 supports effective genome editing in mouse lung airway. Liang, Shun-Qing; Walkey, Christopher J; Martinez, Alexa E; Su, Qin; Dickinson, Mary E; Wang, Dan; Lagor, William R; Heaney, Jason D; Gao, Guangping; Xue, Wen Molecular therapy : the journal of the American Society of Gene Therapy 34695545 2021 Oct 23
Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo. Ibraheim, Raed; Tai, Phillip W L; Mir, Aamir; Javeed, Nida; Wang, Jiaming; Rodríguez, Tomás C; Namkung, Suk; Nelson, Samantha; Khokhar, Eraj Shafiq; Mintzer, Esther; Maitland, Stacy; Chen, Zexiang; Cao, Yueying; Tsagkaraki, Emmanouela; Wolfe, Scot A; Wang, Dan; Pai, Athma A; Xue, Wen; Gao, Guangping; Sontheimer, Erik J Nature communications 34725353 2021 11 01
Gain-of-function cardiomyopathic mutations in RBM20 rewire splicing regulation and re-distribute ribonucleoprotein granules within processing bodies. Fenix, Aidan M; Miyaoka, Yuichiro; Bertero, Alessandro; Blue, Steven M; Spindler, Matthew J; Tan, Kenneth K B; Perez-Bermejo, Juan A; Chan, Amanda H; Mayerl, Steven J; Nguyen, Trieu D; Russell, Caitlin R; Lizarraga, Paweena P; Truong, Annie; So, Po-Lin; Kulkarni, Aishwarya; Chetal, Kashish; Sathe, Shashank; Sniadecki, Nathan J; Yeo, Gene W; Murry, Charles E; Conklin, Bruce R; Salomonis, Nathan Nature communications 34732726 2021 11 03
Disruption of HIV-1 co-receptors CCR5 and CXCR4 in primary human T cells and hematopoietic stem and progenitor cells using base editing. Knipping, Friederike; Newby, Gregory A; Eide, Cindy R; McElroy, Amber N; Nielsen, Sarah C; Smith, Kyle; Fang, Yongxing; Cornu, Tatjana I; Costa, Caroline; Gutierrez-Guerrero, Alejandra; Bingea, Samuel P; Feser, Colby J; Steinbeck, Benjamin; Hippen, Keli L; Blazar, Bruce R; McCaffrey, Anton; Mussolino, Claudio; Verhoeyen, Els; Tolar, Jakub; Liu, David R; Osborn, Mark J Molecular therapy : the journal of the American Society of Gene Therapy 34737067 2022 01 05
Cross-validation of SARS-CoV-2 responses in kidney organoids and clinical populations. Helms, Louisa; Marchiano, Silvia; Stanaway, Ian B; Hsiang, Tien-Ying; Juliar, Benjamin A; Saini, Shally; Zhao, Yan Ting; Khanna, Akshita; Menon, Rajasree; Alakwaa, Fadhl; Mikacenic, Carmen; Morrell, Eric D; Wurfel, Mark M; Kretzler, Matthias; Harder, Jennifer L; Murry, Charles E; Himmelfarb, Jonathan; Ruohola-Baker, Hannele; Bhatraju, Pavan K; Gale Jr, Michael; Freedman, Benjamin S JCI insight 34767537 2021 12 22
Defining genome-wide CRISPR-Cas genome-editing nuclease activity with GUIDE-seq. Malinin, Nikolay L; Lee, GaHyun; Lazzarotto, Cicera R; Li, Yichao; Zheng, Zongli; Nguyen, Nhu T; Liebers, Matthew; Topkar, Ved V; Iafrate, A John; Le, Long P; Aryee, Martin J; Joung, J Keith; Tsai, Shengdar Q Nature protocols 34773119 2021 Nov 12
Neuromuscular Development and Disease: Learning From in vitro and in vivo Models. Fralish, Zachary; Lotz, Ethan M; Chavez, Taylor; Khodabukus, Alastair; Bursac, Nenad Frontiers in cell and developmental biology 34778273 2021
Synthetic biology: at the crossroads of genetic engineering and human therapeutics-a Keystone Symposia report. Cable, Jennifer; Leonard, Joshua N; Lu, Timothy K; Xie, Zhen; Chang, Matthew Wook; Fernández, Luis Ángel; Lora, José M; Kaufman, Howard L; Quintana, Francisco J; Geiger, Roger; F Lesser, Cammie; Lynch, Jason P; Hava, David L; Cornish, Virginia W; Lee, Gary K; DiAndreth, Breanna; Fero, Michael; Srivastava, Rajkamal; De Coster, Tim; Roybal, Kole T; Rackham, Owen J L; Kiani, Samira; Zhu, Iowis; Hernandez-Lopez, Rogelio A; Guo, Tingxi; Chen, William C W Annals of the New York Academy of Sciences 34786712 2021 12
Deleting DNMT3A in CAR T cells prevents exhaustion and enhances antitumor activity. Prinzing, Brooke; Zebley, Caitlin C; Petersen, Christopher T; Fan, Yiping; Anido, Alejandro Allo; Yi, Zhongzhen; Nguyen, Phuong; Houke, Haley; Bell, Matthew; Haydar, Dalia; Brown, Charmaine; Boi, Shannon K; Alli, Shanta; Crawford, Jeremy Chase; Riberdy, Janice M; Park, Jeoungeun J; Zhou, Sheng; Velasquez, Mireya Paulina; DeRenzo, Chris; Lazzarotto, Cicera R; Tsai, Shengdar Q; Vogel, Peter; Pruett-Miller, Shondra M; Langfitt, Deanna M; Gottschalk, Stephen; Youngblood, Ben; Krenciute, Giedre Science translational medicine 34788079 2021 Nov 17
Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models. Hakim, Chady H; Kumar, Sandeep R P; Pérez-López, Dennis O; Wasala, Nalinda B; Zhang, Dong; Yue, Yongping; Teixeira, James; Pan, Xiufang; Zhang, Keqing; Million, Emily D; Nelson, Christopher E; Metzger, Samantha; Han, Jin; Louderman, Jacqueline A; Schmidt, Florian; Feng, Feng; Grimm, Dirk; Smith, Bruce F; Yao, Gang; Yang, N Nora; Gersbach, Charles A; Chen, Shi-Jie; Herzog, Roland W; Duan, Dongsheng Nature communications 34819506 2021 11 24
CRISPR-enhanced human adipocyte browning as cell therapy for metabolic disease. Tsagkaraki, Emmanouela; Nicoloro, Sarah M; DeSouza, Tiffany; Solivan-Rivera, Javier; Desai, Anand; Lifshitz, Lawrence M; Shen, Yuefei; Kelly, Mark; Guilherme, Adilson; Henriques, Felipe; Amrani, Nadia; Ibraheim, Raed; Rodriguez, Tomas C; Luk, Kevin; Maitland, Stacy; Friedline, Randall H; Tauer, Lauren; Hu, Xiaodi; Kim, Jason K; Wolfe, Scot A; Sontheimer, Erik J; Corvera, Silvia; Czech, Michael P Nature communications 34836963 2021 11 26
The FusX TALE Base Editor (FusXTBE) for Rapid Mitochondrial DNA Programming of Human Cells In Vitro and Zebrafish Disease Models In Vivo. Sabharwal, Ankit; Kar, Bibekananda; Restrepo-Castillo, Santiago; Holmberg, Shannon R; Mathew, Neal D; Kendall, Benjamin Luke; Cotter, Ryan P; WareJoncas, Zachary; Seiler, Christoph; Nakamaru-Ogiso, Eiko; Clark, Karl J; Ekker, Stephen C The CRISPR journal 34847747 2021 12
The Coiled-Coil Forming Peptide (KVSALKE)5 Is a Cell Penetrating Peptide that Enhances the Intracellular Delivery of Proteins. Li, Jie; Tuma, Jan; Han, Hesong; Kim, Hansol; Wilson, Ross C; Lee, Hye Young; Murthy, Niren Advanced healthcare materials 34861744 2021 Dec 03
Author Correction: Engineered pegRNAs improve prime editing efficiency. Nelson, James W; Randolph, Peyton B; Shen, Simon P; Everette, Kelcee A; Chen, Peter J; Anzalone, Andrew V; An, Meirui; Newby, Gregory A; Chen, Jonathan C; Hsu, Alvin; Liu, David R Nature biotechnology 34880468 2021 Dec 08
Programmable deletion, replacement, integration and inversion of large DNA sequences with twin prime editing. Anzalone, Andrew V; Gao, Xin D; Podracky, Christopher J; Nelson, Andrew T; Koblan, Luke W; Raguram, Aditya; Levy, Jonathan M; Mercer, Jaron A M; Liu, David R Nature biotechnology 34887556 2021 Dec 09
AAV capsid variants with brain-wide transgene expression and decreased liver targeting after intravenous delivery in mouse and marmoset. Goertsen, David; Flytzanis, Nicholas C; Goeden, Nick; Chuapoco, Miguel R; Cummins, Alexander; Chen, Yijing; Fan, Yingying; Zhang, Qiangge; Sharma, Jitendra; Duan, Yangyang; Wang, Liping; Feng, Guoping; Chen, Yu; Ip, Nancy Y; Pickel, James; Gradinaru, Viviana Nature neuroscience 34887588 2022 01
Breast cancer immunotherapy: current biomarkers and the potential of in vitro assays. Skala, Melissa C; Ayuso, Jose M; Burkard, Mark E; Deming, Dustin A Current opinion in biomedical engineering 34901585 2021 Mar
Reading Frame Repair of TTN Truncation Variants Restores Titin Quantity and Functions. Romano, Robert; Ghahremani, Shahnaz; Zimmerman, Talia; Legere, Nicholas; Thakar, Ketan; Ladha, Feria A; Pettinato, Anthony M; Hinson, J Travis Circulation 34905694 2022 01 18
Acid-Sensitive Surfactants Enhance the Delivery of Nucleic Acids. Røise, Joachim Justad; Han, Hesong; Li, Jie; Kerr, D Lucas; Taing, Chung; Behrouzi, Kamyar; He, Maomao; Ruan, Emily; Chan, Lienna Y; Espinoza, Eli M; Reinhard, Sören; Thakker, Kanav; Kwon, Justin; Mofrad, Mohammad R K; Murthy, Niren Molecular pharmaceutics 34931518 2022 01 03
CRISPR/Cas9 ribonucleoprotein-mediated genome and epigenome editing in mammalian cells. Advanced drug delivery reviews 34942274 2022 Feb
rAAV-delivered PTEN therapeutics for prostate cancer. Ai, Jianzhong; Li, Jia; Su, Qin; Ma, Hong; Wei, Qiang; Li, Hong; Gao, Guangping Molecular therapy. Nucleic acids 34976432 2022 Mar 08
Editorial: "AAV Gene Therapy: Immunology and Immunotherapeutics". Martinez-Navio, José M; Paulk, Nicole K; Gao, Guangping Frontiers in immunology 34992614 2021
A flexible split prime editor using truncated reverse transcriptase improves dual-AAV delivery in mouse liver. Zheng, Chunwei; Liang, Shun-Qing; Liu, Bin; Liu, Pengpeng; Kwan, Suet-Yan; Wolfe, Scot A; Xue, Wen Molecular therapy : the journal of the American Society of Gene Therapy 34998953 2022 Jan 05
Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins. Banskota, Samagya; Raguram, Aditya; Suh, Susie; Du, Samuel W; Davis, Jessie R; Choi, Elliot H; Wang, Xiao; Nielsen, Sarah C; Newby, Gregory A; Randolph, Peyton B; Osborn, Mark J; Musunuru, Kiran; Palczewski, Krzysztof; Liu, David R Cell 35021064 2022 01 20
BRD2 inhibition blocks SARS-CoV-2 infection by reducing transcription of the host cell receptor ACE2. Samelson, Avi J; Tran, Quang Dinh; Robinot, Rémy; Carrau, Lucia; Rezelj, Veronica V; Kain, Alice Mac; Chen, Merissa; Ramadoss, Gokul N; Guo, Xiaoyan; Lim, Shion A; Lui, Irene; Nuñez, James K; Rockwood, Sarah J; Wang, Jianhui; Liu, Na; Carlson-Stevermer, Jared; Oki, Jennifer; Maures, Travis; Holden, Kevin; Weissman, Jonathan S; Wells, James A; Conklin, Bruce R; TenOever, Benjamin R; Chakrabarti, Lisa A; Vignuzzi, Marco; Tian, Ruilin; Kampmann, Martin Nature cell biology 35027731 2022 01
Detecting Cardiovascular Protein-Protein Interactions by Proximity Proteomics. Circulation research 35050691 2022 01 21
Genome-wide detection of CRISPR editing in vivo using GUIDE-tag. Liang, Shun-Qing; Liu, Pengpeng; Smith, Jordan L; Mintzer, Esther; Maitland, Stacy; Dong, Xiaolong; Yang, Qiyuan; Lee, Jonathan; Haynes, Cole M; Zhu, Lihua Julie; Watts, Jonathan K; Sontheimer, Erik J; Wolfe, Scot A; Xue, Wen Nature communications 35064134 2022 01 21
pH-Responsive Polymer Nanoparticles for Efficient Delivery of Cas9 Ribonucleoprotein With or Without Donor DNA. Xie, Ruosen; Wang, Xiuxiu; Wang, Yuyuan; Ye, Mingzhou; Zhao, Yi; Yandell, Brian S; Gong, Shaoqin Advanced materials (Deerfield Beach, Fla.) 35119139 2022 Feb 04
Phage Display-Derived Peptide for the Specific Binding of SARS-CoV-2. Yang, Fan; Liu, Li; Neuenschwander, Pierre Fernand; Idell, Steven; Vankayalapati, Ramakrishna; Jain, Krishan Gopal; Du, Ke; Ji, Honglong; Yi, Guohua ACS omega 35128233 2022 Feb 01
Species-dependent in vivo mRNA delivery and cellular responses to nanoparticles. Hatit, Marine Z C; Lokugamage, Melissa P; Dobrowolski, Curtis N; Paunovska, Kalina; Ni, Huanzhen; Zhao, Kun; Vanover, Daryll; Beyersdorf, Jared; Peck, Hannah E; Loughrey, David; Sato, Manaka; Cristian, Ana; Santangelo, Philip J; Dahlman, James E Nature nanotechnology 35132167 2022 Mar
A Human-derived Dual MRI/PET Reporter Gene System with High Translational Potential for Cell Tracking. Shalaby, Nourhan; Kelly, John; Martinez, Francisco; Fox, Mathew; Qi, Qi; Thiessen, Jonathan; Hicks, Justin; Scholl, Timothy J; Ronald, John A Molecular imaging and biology 35146614 2022 04
Nonhuman Primates in Translational Research. Tarantal, Alice F; Noctor, Stephen C; Hartigan-O'Connor, Dennis J Annual review of animal biosciences 35167321 2022 02 15
A self-immolative linker that releases thiols detects penicillin amidase and nitroreductase with high sensitivity via absorption spectroscopy. Espinoza, Eli M; Røise, Joachim J; He, Maomao; Li, I-Che; Agatep, Alvin K; Udenyi, Patrick; Han, Hesong; Jackson, Nicole; Kerr, D Lucas; Chen, Dake; Stentzel, Michael R; Ruan, Emily; Riley, Lee; Murthy, Niren Chemical communications (Cambridge, England) 35170593 2022 Mar 03
Intestinal Deletion of 3-Hydroxy-3-Methylglutaryl-Coenzyme A Reductase Promotes Expansion of the Resident Stem Cell Compartment. Doerfler, Alexandria M; Han, Jun; Jarrett, Kelsey E; Tang, Li; Jain, Antrix; Saltzman, Alexander; De Giorgi, Marco; Chuecos, Marcel; Hurley, Ayrea E; Li, Ang; Morand, Pauline; Ayala, Claudia; Goodlett, David R; Malovannaya, Anna; Martin, James F; de Aguiar Vallim, Thomas Q; Shroyer, Noah; Lagor, William R Arteriosclerosis, thrombosis, and vascular biology 35172604 2022 Apr
Lung-selective mRNA delivery of synthetic lipid nanoparticles for the treatment of pulmonary lymphangioleiomyomatosis. Qiu, Min; Tang, Yan; Chen, Jinjin; Muriph, Rachel; Ye, Zhongfeng; Huang, Changfeng; Evans, Jason; Henske, Elizabeth P; Xu, Qiaobing Proceedings of the National Academy of Sciences of the United States of America 35173043 2022 02 22
Derivation of extra-embryonic and intra-embryonic macrophage lineages from human pluripotent stem cells. Bredemeyer, Andrea L; Amrute, Junedh M; Koenig, Andrew L; Idol, Rachel A; He, Li; Luff, Stephanie A; Dege, Carissa; Leid, Jamison M; Schilling, Joel D; Hinson, J Travis; Dinauer, Mary C; Sturgeon, Christopher M; Lavine, Kory J Development (Cambridge, England) 35178561 2022 04 15
Transcription factor protein interactomes reveal genetic determinants in heart disease. Gonzalez-Teran, Barbara; Pittman, Maureen; Felix, Franco; Thomas, Reuben; Richmond-Buccola, Desmond; Hüttenhain, Ruth; Choudhary, Krishna; Moroni, Elisabetta; Costa, Mauro W; Huang, Yu; Padmanabhan, Arun; Alexanian, Michael; Lee, Clara Youngna; Maven, Bonnie E J; Samse-Knapp, Kaitlen; Morton, Sarah U; McGregor, Michael; Gifford, Casey A; Seidman, J G; Seidman, Christine E; Gelb, Bruce D; Colombo, Giorgio; Conklin, Bruce R; Black, Brian L; Bruneau, Benoit G; Krogan, Nevan J; Pollard, Katherine S; Srivastava, Deepak Cell 35182466 2022 03 03
Rapid, accurate mapping of transgene integration in viable rhesus macaque embryos using enhanced-specificity tagmentation-assisted PCR. Ryu, Junghyun; Chan, William; Wettengel, Jochen M; Hanna, Carol B; Burwitz, Benjamin J; Hennebold, Jon D; Bimber, Benjamin N Molecular therapy. Methods & clinical development 35211637 2022 Mar 10
Magnetic Iron Oxide Nanoparticles for Biomedical Applications. Jiang, Kaiyi; Zhang, Linlin; Bao, Gang Current opinion in biomedical engineering 35211662 2021 Dec
Treating Cardiovascular Disease with Liver Genome Engineering. Hurley, Ayrea; Lagor, William R Current atherosclerosis reports 35230602 2022 Feb
Modeling genetic diseases in nonhuman primates through embryonic and germline modification: Considerations and challenges. Schmidt, Jenna K; Jones, Kathryn M; Van Vleck, Trevor; Emborg, Marina E Science translational medicine 35235338 2022 03 02
Modeling injury and repair in kidney organoids reveals that homologous recombination governs tubular intrinsic repair. Gupta, Navin; Matsumoto, Takuya; Hiratsuka, Ken; Garcia Saiz, Edgar; Galichon, Pierre; Miyoshi, Tomoya; Susa, Koichiro; Tatsumoto, Narihito; Yamashita, Michifumi; Morizane, Ryuji Science translational medicine 35235339 2022 Mar 02
Antispacer peptide nucleic acids for sequence-specific CRISPR-Cas9 modulation. Economos, Nicholas G; Quijano, Elias; Carufe, Kelly E W; Perera, J Dinithi R; Glazer, Peter M Nucleic acids research 35235944 2022 Mar 03
In Vivo Gene Editing in Lipid and Atherosclerosis Research. De Giorgi, Marco; Jarrett, Kelsey E; de Aguiar Vallim, Thomas Q; Lagor, William R Methods in molecular biology (Clifton, N.J.) 35237996 2022
Clinical magnetic hyperthermia requires integrated magnetic particle imaging. Healy, Sean; Bakuzis, Andris F; Goodwill, Patrick W; Attaluri, Anilchandra; Bulte, Jeff W M; Ivkov, Robert Wiley interdisciplinary reviews. Nanomedicine and nanobiotechnology 35238181 2022 May
Design of therapeutic biomaterials to control inflammation. Tu, Zhaoxu; Zhong, Yiling; Hu, Hanze; Shao, Dan; Haag, Rainer; Schirner, Michael; Lee, Jaewoo; Sullenger, Bruce; Leong, Kam W Nature reviews. Materials 35251702 2022 Feb 28
Technologies to Assess Drug Response and Heterogeneity in Patient-Derived Cancer Organoids. Skala, Melissa C; Deming, Dustin A; Kratz, Jeremy D Annual review of biomedical engineering 35259932 2022 Feb 08
Durability of transgene expression after rAAV gene therapy. Muhuri, Manish; Levy, Daniel I; Schulz, Martin; McCarty, Douglas; Gao, Guangping Molecular therapy : the journal of the American Society of Gene Therapy 35283274 2022 04 06
Translational Utility of the Nonhuman Primate Model. Tarantal, Alice F; Hartigan-O'Connor, Dennis J; Noctor, Stephen C Biological psychiatry. Cognitive neuroscience and neuroimaging 35283343 2022 05
Human and Insect Cell-Produced Recombinant Adeno-Associated Viruses Show Differences in Genome Heterogeneity. Tran, Ngoc Tam; Lecomte, Emilie; Saleun, Sylvie; Namkung, Suk; Robin, Cécile; Weber, Kristina; Devine, Eric; Blouin, Veronique; Adjali, Oumeya; Ayuso, Eduard; Gao, Guangping; Penaud-Budloo, Magalie; Tai, Phillip W L Human gene therapy 35293222 2022 04
Toward improved understanding of cardiac development and congenital heart disease: The advent of cardiac organoids. Scherba, Jacob C; Karra, Ravi; Turek, Joseph W; Bursac, Nenad The Journal of thoracic and cardiovascular surgery 35307217 2022 Dec
AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice. Wang, Jiaming; Zhang, Yue; Mendonca, Craig A; Yukselen, Onur; Muneeruddin, Khaja; Ren, Lingzhi; Liang, Jialing; Zhou, Chen; Xie, Jun; Li, Jia; Jiang, Zhong; Kucukural, Alper; Shaffer, Scott A; Gao, Guangping; Wang, Dan Nature 35322228 2022 04
Bioengineered Kidney Models: Methods and Functional Assessments. Rizki-Safitri, Astia; Traitteur, Tamara; Morizane, Ryuji Function (Oxford, England) 35330622 2021
Impact of baseline culture conditions of cancer organoids when determining therapeutic response and tumor heterogeneity. DeStefanis, Rebecca A; Kratz, Jeremy D; Olson, Autumn M; Sunil, Aishwarya; DeZeeuw, Alyssa K; Gillette, Amani A; Sha, Gioia C; Johnson, Katherine A; Pasch, Cheri A; Clipson, Linda; Skala, Melissa C; Deming, Dustin A Scientific reports 35338174 2022 03 25
Robust, Durable Gene Activation In Vivo via mRNA-Encoded Activators. Beyersdorf, Jared P; Bawage, Swapnil; Iglesias, Nahid; Peck, Hannah E; Hobbs, Ryan A; Wroe, Jay A; Zurla, Chiara; Gersbach, Charles A; Santangelo, Philip J ACS nano 35357116 2022 Mar 31
Efficient targeted transgenesis of large donor DNA into multiple mouse genetic backgrounds using bacteriophage Bxb1 integrase. Low, Benjamin E; Hosur, Vishnu; Lesbirel, Simon; Wiles, Michael V Scientific reports 35361849 2022 03 31
Physiology assays in human kidney organoids. Freedman, Benjamin S American journal of physiology. Renal physiology 35379001 2022 06 01
CRISPR-free base editors with enhanced activity and expanded targeting scope in mitochondrial and nuclear DNA. Mok, Beverly Y; Kotrys, Anna V; Raguram, Aditya; Huang, Tony P; Mootha, Vamsi K; Liu, David R Nature biotechnology 35379961 2022 Apr 04
A split prime editor with untethered reverse transcriptase and circular RNA template. Liu, Bin; Dong, Xiaolong; Cheng, Haoyang; Zheng, Chunwei; Chen, Zexiang; Rodríguez, Tomás C; Liang, Shun-Qing; Xue, Wen; Sontheimer, Erik J Nature biotechnology 35379962 2022 09
In vivo base editing rescues cone photoreceptors in a mouse model of early-onset inherited retinal degeneration. Choi, Elliot H; Suh, Susie; Foik, Andrzej T; Leinonen, Henri; Newby, Gregory A; Gao, Xin D; Banskota, Samagya; Hoang, Thanh; Du, Samuel W; Dong, Zhiqian; Raguram, Aditya; Kohli, Sajeev; Blackshaw, Seth; Lyon, David C; Liu, David R; Palczewski, Krzysztof Nature communications 35383196 2022 Apr 05
Surface conjugation of antibodies improves nanoparticle uptake in bronchial epithelial cells. Luks, Valerie L; Mandl, Hanna; DiRito, Jenna; Barone, Christina; Freedman-Weiss, Mollie R; Ricciardi, Adele S; Tietjen, Gregory G; Egan, Marie E; Saltzman, W Mark; Stitelman, David H PloS one 35385514 2022
Multivalent designed proteins neutralize SARS-CoV-2 variants of concern and confer protection against infection in mice. Hunt, Andrew C; Case, James Brett; Park, Young-Jun; Cao, Longxing; Wu, Kejia; Walls, Alexandra C; Liu, Zhuoming; Bowen, John E; Yeh, Hsien-Wei; Saini, Shally; Helms, Louisa; Zhao, Yan Ting; Hsiang, Tien-Ying; Starr, Tyler N; Goreshnik, Inna; Kozodoy, Lisa; Carter, Lauren; Ravichandran, Rashmi; Green, Lydia B; Matochko, Wadim L; Thomson, Christy A; Vögeli, Bastian; Krüger, Antje; VanBlargan, Laura A; Chen, Rita E; Ying, Baoling; Bailey, Adam L; Kafai, Natasha M; Boyken, Scott E; Ljubetič, Ajasja; Edman, Natasha; Ueda, George; Chow, Cameron M; Johnson, Max; Addetia, Amin; Navarro, Mary Jane; Panpradist, Nuttada; Gale Jr, Michael; Freedman, Benjamin S; Bloom, Jesse D; Ruohola-Baker, Hannele; Whelan, Sean P J; Stewart, Lance; Diamond, Michael S; Veesler, David; Jewett, Michael C; Baker, David Science translational medicine 35412328 2022 Apr 12
Myoblast deactivation within engineered human skeletal muscle creates a transcriptionally heterogeneous population of quiescent satellite-like cells. Wang, Jason; Broer, Torie; Chavez, Taylor; Zhou, Chris J; Tran, Sabrina; Xiang, Yu; Khodabukus, Alastair; Diao, Yarui; Bursac, Nenad Biomaterials 35421801 2022 05
CRISPR-Cas9 bends and twists DNA to read its sequence. Cofsky, Joshua C; Soczek, Katarzyna M; Knott, Gavin J; Nogales, Eva; Doudna, Jennifer A Nature structural & molecular biology 35422516 2022 Apr
Crystal structure of an RNA/DNA strand exchange junction. Cofsky, Joshua C; Knott, Gavin J; Gee, Christine L; Doudna, Jennifer A PloS one 35436289 2022
Tetrazine-Ligated CRISPR sgRNAs for Efficient Genome Editing. Chen, Zexiang; Devi, Gitali; Arif, Amena; Zamore, Phillip D; Sontheimer, Erik J; Watts, Jonathan K ACS chemical biology 35446558 2022 05 20
Modelling ciliopathy phenotypes in human tissues derived from pluripotent stem cells with genetically ablated cilia. Cruz, Nelly M; Reddy, Raghava; McFaline-Figueroa, José L; Tran, Christine; Fu, Hongxia; Freedman, Benjamin S Nature biomedical engineering 35478224 2022 04
An optimized FusX assembly-based technique to introduce mitochondrial TC-to-TT variations in human cell lines. Kar, Bibekananda; Sabharwal, Ankit; Restrepo-Castillo, Santiago; Simone, Brandon W; Clark, Karl J; Ekker, Stephen C STAR protocols 35496789 2022 Jun 17
Identification and Validation of CRISPR/Cas9 Off-Target Activity in Hematopoietic Stem and Progenitor Cells. Park, So Hyun; Lee, Ciaran M; Bao, Gang Methods in molecular biology (Clifton, N.J.) 35507169 2022
Prioritization of autoimmune disease-associated genetic variants that perturb regulatory element activity in T cells. Mouri, Kousuke; Guo, Michael H; de Boer, Carl G; Lissner, Michelle M; Harten, Ingrid A; Newby, Gregory A; DeBerg, Hannah A; Platt, Winona F; Gentili, Matteo; Liu, David R; Campbell, Daniel J; Hacohen, Nir; Tewhey, Ryan; Ray, John P Nature genetics 35513721 2022 05
The clinical progress of mRNA vaccines and immunotherapies. Barbier, Ann J; Jiang, Allen Yujie; Zhang, Peng; Wooster, Richard; Anderson, Daniel G Nature biotechnology 35534554 2022 06
Structural biology of CRISPR-Cas immunity and genome editing enzymes. Wang, Joy Y; Pausch, Patrick; Doudna, Jennifer A Nature reviews. Microbiology 35562427 2022 11
A High-Efficiency AAV for Endothelial Cell Transduction Throughout the Central Nervous System. Krolak, Trevor; Chan, Ken Y; Kaplan, Luke; Huang, Qin; Wu, Jason; Zheng, Qingxia; Kozareva, Velina; Beddow, Thomas; Tobey, Isabelle G; Pacouret, Simon; Chen, Albert T; Chan, Yujia A; Ryvkin, Daniel; Gu, Chenghua; Deverman, Benjamin E Nature cardiovascular research 35571675 2022 Apr
Label-Free Imaging to Track Reprogramming of Human Somatic Cells. Molugu, Kaivalya; Battistini, Giovanni A; Heaster, Tiffany M; Rouw, Jacob; Guzman, Emmanuel C; Skala, Melissa C; Saha, Krishanu GEN biotechnology 35586336 2022 Apr 01
CRISPR Library Screening in Cultured Cardiomyocytes. DeLuca, Sophia; Bursac, Nenad Methods in molecular biology (Clifton, N.J.) 35618895 2022
Imaging the fetal nonhuman primate brain with SV2A positron emission tomography (PET). Rossano, Samantha; Toyonaga, Takuya; Berg, Eric; Lorence, Isabella; Fowles, Krista; Nabulsi, Nabeel; Ropchan, Jim; Li, Songye; Ye, Yunpeng; Felchner, Zachary; Kukis, David; Huang, Yiyun; Benveniste, Helene; Tarantal, Alice F; Groman, Stephanie; Carson, Richard E European journal of nuclear medicine and molecular imaging 35633376 2022 09
Inhibition of B-cell lymphoma 2 family proteins alters optical redox ratio, mitochondrial polarization, and cell energetics independent of cell state. Gillette, Amani A; DeStefanis, Rebecca A; Pritzl, Stephanie L; Deming, Dustin A; Skala, Melissa C Journal of biomedical optics 35643815 2022 05
Synthesis and ex vivo biodistribution of two 68Ga-labeled tetrazine tracers: Comparison of pharmacokinetics. Lambidis, Elisavet; Lumén, Dave; Koskipahta, Elina; Imlimthan, Surachet; Lopez, Brianda B; Sánchez, Ana Isabel Fraguas; Sarparanta, Mirkka; Cheng, R Holland; Airaksinen, Anu J Nuclear medicine and biology 35680503 2022
In vivo engineered B cells secrete high titers of broadly neutralizing anti-HIV antibodies in mice. Nahmad, Alessio D; Lazzarotto, Cicera R; Zelikson, Natalie; Kustin, Talia; Tenuta, Mary; Huang, Deli; Reuveni, Inbal; Nataf, Daniel; Raviv, Yuval; Horovitz-Fried, Miriam; Dotan, Iris; Carmi, Yaron; Rosin-Arbesfeld, Rina; Nemazee, David; Voss, James E; Ste Nature biotechnology 35681059 2022 Aug
The balance between protective and pathogenic immune responses to pneumonia in the neonatal lung is enforced by gut microbiota. Stevens, Joseph; Steinmeyer, Shelby; Bonfield, Madeline; Peterson, Laura; Wang, Timothy; Gray, Jerilyn; Lewkowich, Ian; Xu, Yan; Du, Yina; Guo, Minzhe; Wynn, James L; Zacharias, William; Salomonis, Nathan; Miller, Lisa; Chougnet, Claire; O'Connor, Dennis Hartigan; Deshmukh, Hitesh Science translational medicine 35704600 2022 Jun 15
Efficient Genome Editing Achieved via Plug-and-Play Adenovirus Piggyback Transport of Cas9/gRNA Complex on Viral Capsid Surface. Lu, Zhi Hong; Li, Jie; Dmitriev, Igor P; Kashentseva, Elena A; Curiel, David T ACS nano 35749339 2022 Jun 24
Kidney organoids: a pioneering model for kidney diseases. Tekguc, Murat; Gaal, Ronald C VAN; Uzel, Sebastien G M; Gupta, Navin; Riella, Leonardo V; Lewis, Jennifer A; Morizane, Ryuji Translational research : the journal of laboratory and clinical medicine 35750295 2022 Jun 21
Nanoparticle single-cell multiomic readouts reveal that cell heterogeneity influences lipid nanoparticle-mediated messenger RNA delivery. Dobrowolski, Curtis; Paunovska, Kalina; Schrader Echeverri, Elisa; Loughrey, David; Da Silva Sanchez, Alejandro J; Ni, Huanzhen; Hatit, Marine Z C; Lokugamage, Melissa P; Kuzminich, Yanina; Peck, Hannah E; Santangelo, Philip J; Dahlman, James E Nature nanotechnology 35768613 2022 08
In situ autofluorescence lifetime assay of a photoreceptor stimulus response in mouse retina and human retinal organoids. Samimi, Kayvan; Pattnaik, Bikash R; Capowski, Elizabeth E; Saha, Krishanu; Gamm, David M; Skala, Melissa C Biomedical optics express 35781966 2022 Jun 01
Therapeutic in vivo delivery of gene editing agents. Raguram, Aditya; Banskota, Samagya; Liu, David R Cell 35798006 2022 Jul 21
SARS-CoV-2 Host Immunogenetic Biomarkers. Gededzha, Maemu P; Mampeule, Nakampe; Gandini, Anastasia; Mayne, Elizabeth S Methods in molecular biology (Clifton, N.J.) 35838957 2022
Evaluation Protocol for SARS-CoV-2 Serological Assays. Gededzha, Maemu P; Jugwanth, Sarika; Mampeule, Nakampe; Zwane, Nontobeko; David, Anura; Scott, Lesley; Stevens, Wendy; Mayne, Elizabeth S Methods in molecular biology (Clifton, N.J.) 35838970 2022
Non-Invasive imaging of extracellular vesicles: Quo vaditis in vivo? Arifin, Dian R; Witwer, Kenneth W; Bulte, Jeff W M Journal of extracellular vesicles 35844061 2022 Jul
Development of 68Ga-Labeled Hepatitis E Virus Nanoparticles for Targeted Drug Delivery and Diagnostics with PET. Lambidis, Elisavet; Chen, Chun-Chieh; Baikoghli, Mo; Imlimthan, Surachet; Khng, You Cheng; Sarparanta, Mirkka; Cheng, R Holland; Airaksinen, Anu J Molecular pharmaceutics 35857429 2022 08 01
Efficient in vivo base editing via single adeno-associated viruses with size-optimized genomes encoding compact adenine base editors. Davis, Jessie R; Wang, Xiao; Witte, Isaac P; Huang, Tony P; Levy, Jonathan M; Raguram, Aditya; Banskota, Samagya; Seidah, Nabil G; Musunuru, Kiran; Liu, David R Nature biomedical engineering 35902773 2022 Jul 28
Multiomics characterization of mouse hepatoblastoma identifies yes-associated protein 1 target genes. Rodríguez, Tomás C; Kwan, Suet-Yan; Smith, Jordan L; Dadafarin, Sina; Wu, Chern-Horng; Sontheimer, Erik J; Xue, Wen Hepatology (Baltimore, Md.) 35932276 2022 Aug 06
Designing and executing prime editing experiments in mammalian cells. Doman, Jordan L; Sousa, Alexander A; Randolph, Peyton B; Chen, Peter J; Liu, David R Nature protocols 35941224 2022 Aug 08
Material Engineering in Gut Microbiome and Human Health. Yang, Letao; Hung, Lin Y; Zhu, Yuefei; Ding, Suwan; Margolis, Kara G; Leong, Kam W Research (Washington, D.C.) 35958108 2022
Piperazine-derived lipid nanoparticles deliver mRNA to immune cells in vivo. Ni, Huanzhen; Hatit, Marine Z C; Zhao, Kun; Loughrey, David; Lokugamage, Melissa P; Peck, Hannah E; Cid, Ada Del; Muralidharan, Abinaya; Kim, YongTae; Santangelo, Philip J; Dahlman, James E Nature communications 35970837 2022 08 15
Prostate cancer cells demonstrate unique metabolism and substrate adaptability acutely after androgen deprivation therapy. Filon, Mikolaj J; Gillette, Amani A; Yang, Bing; Khemees, Tariq A; Skala, Melissa C; Jarrard, David F The Prostate 35980831 2022 Dec
An ELISA-based platform for rapid identification of structure-dependent nucleic acid-protein interactions detects novel DNA triplex interactors. Economos, Nicholas G; Thapar, Upasna; Balasubramanian, Nanda; Karras, Georgios I; Glazer, Peter M The Journal of biological chemistry 35988651 2022 Oct
High-throughput continuous evolution of compact Cas9 variants targeting single-nucleotide-pyrimidine PAMs. Huang, Tony P; Heins, Zachary J; Miller, Shannon M; Wong, Brandon G; Balivada, Pallavi A; Wang, Tina; Khalil, Ahmad S; Liu, David R Nature biotechnology 36076084 2022 Sep 08
Differential microRNA profiles of intramuscular and secreted extracellular vesicles in human tissue-engineered muscle. Vann, Christopher G; Zhang, Xin; Khodabukus, Alastair; Orenduff, Melissa C; Chen, Yu-Hsiu; Corcoran, David L; Truskey, George A; Bursac, Nenad; Kraus, Virginia B Frontiers in physiology 36091396 2022
Wnt5a/β-catenin axis is involved in the downregulation of AT2 lineage by PAI-1. Jain, Krishan G; Zhao, Runzhen; Liu, Yang; Guo, Xuan; Yi, Guohua; Ji, Hong-Long American journal of physiology. Lung cellular and molecular physiology 36098461 2022 Nov 01
Precision genome editing in the eye. Suh, Susie; Choi, Elliot H; Raguram, Aditya; Liu, David R; Palczewski, Krzysztof Proceedings of the National Academy of Sciences of the United States of America 36122230 2022 Sep 27
Promoting validation and cross-phylogenetic integration in model organism research. Cheng, Keith C; Burdine, Rebecca D; Dickinson, Mary E; Ekker, Stephen C; Lin, Alex Y; Lloyd, K C Kent; Lutz, Cathleen M; MacRae, Calum A; Morrison, John H; O'Connor, David H; Postlethwait, John H; Rogers, Crystal D; Sanchez, Susan; Simpson, Julie H; Talbot, William S; Wallace, Douglas C; Weimer, Jill M; Bellen, Hugo J Disease models & mechanisms 36125045 2022 09 01
Organoid-on-a-chip model of human ARPKD reveals mechanosensing pathomechanisms for drug discovery. Hiratsuka, Ken; Miyoshi, Tomoya; Kroll, Katharina T; Gupta, Navin R; Valerius, M Todd; Ferrante, Thomas; Yamashita, Michifumi; Lewis, Jennifer A; Morizane, Ryuji Science advances 36129975 2022 09 23
Engineering multicellular living systems-A Keystone Symposia report. Cable, Jennifer; Arlotta, Paola; Parker, Kevin Kit; Hughes, Alex J; Goodwin, Katharine; Mummery, Christine L; Kamm, Roger D; Engle, Sandra J; Tagle, Danilo A; Boj, Sylvia F; Stanton, Alice E; Morishita, Yoshihiro; Kemp, Melissa L; Norfleet, Dennis A; May, Annals of the New York Academy of Sciences 36177947 2022 Sep 30
Direct ITR-to-ITR Nanopore Sequencing of AAV Vector Genomes. Namkung, Suk; Tran, Ngoc Tam; Manokaran, Sangeetha; He, Ran; Su, Qin; Xie, Jun; Gao, Guangping; Tai, Phillip W L Human gene therapy 36178359 2022 Nov
In vivo correction of cystic fibrosis mediated by PNA nanoparticles. Piotrowski-Daspit, Alexandra S; Barone, Christina; Lin, Chun-Yu; Deng, Yanxiang; Wu, Douglas; Binns, Thomas C; Xu, Emily; Ricciardi, Adele S; Putman, Rachael; Garrison, Alannah; Nguyen, Richard; Gupta, Anisha; Fan, Rong; Glazer, Peter M; Saltzman, W Mark; Egan, Marie E Science advances 36197984 2022 Oct 07
Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing. Gonzalez, Trevor J; Simon, Katherine E; Blondel, Leo O; Fanous, Marco M; Roger, Angela L; Maysonet, Maribel Santiago; Devlin, Garth W; Smith, Timothy J; Oh, Daniel K; Havlik, L Patrick; Castellanos Rivera, Ruth M; Piedrahita, Jorge A; ElMallah, Mai K; Ger Nature communications 36210364 2022 Oct 10
EZ Clear for simple, rapid, and robust mouse whole organ clearing. Hsu, Chih-Wei; Cerda, Juan; Kirk, Jason M; Turner, Williamson D; Rasmussen, Tara L; Flores Suarez, Carlos P; Dickinson, Mary E; Wythe, Joshua D eLife 36218247 2022 Oct 11
Monobody adapter for functional antibody display on nanoparticles for adaptable targeted delivery applications. Albert, C; Bracaglia, L; Koide, A; DiRito, J; Lysyy, T; Harkins, L; Edwards, C; Richfield, O; Grundler, J; Zhou, K; Denbaum, E; Ketavarapu, G; Hattori, T; Perincheri, S; Langford, J; Feizi, A; Haakinson, D; Hosgood, S A; Nicholson, M L; Pober, J S; Saltzman, W M; Koide, S; Tietjen, G T Nature communications 36220817 2022 Oct 11
Translating musculoskeletal bioengineering into tissue regeneration therapies. Khodabukus, Alastair; Guyer, Tyler; Moore, Axel C; Stevens, Molly M; Guldberg, Robert E; Bursac, Nenad Science translational medicine 36223445 2022 Oct 12
Nucleic Acid Delivery to the Vascular Endothelium. Reschke, Melanie; Piotrowski-Daspit, Alexandra S; Pober, Jordan S; Saltzman, W Mark Molecular pharmaceutics 36251765 2022 Dec 05
Unadjuvanted intranasal spike vaccine elicits protective mucosal immunity against sarbecoviruses. Mao, Tianyang; Israelow, Benjamin; Peña-Hernández, Mario A; Suberi, Alexandra; Zhou, Liqun; Luyten, Sophia; Reschke, Melanie; Dong, Huiping; Homer, Robert J; Saltzman, W Mark; Iwasaki, Akiko Science (New York, N.Y.) 36302057 2022 Nov 25
Adult human kidney organoids originate from CD24+ cells and represent an advanced model for adult polycystic kidney disease. Xu, Yaoxian; Kuppe, Christoph; Perales-Patón, Javier; Hayat, Sikander; Kranz, Jennifer; Abdallah, Ali T; Nagai, James; Li, Zhijian; Peisker, Fabian; Saritas, Turgay; Halder, Maurice; Menzel, Sylvia; Hoeft, Konrad; Kenter, Annegien; Kim, Hyojin; van Roeyen, Claudia R C; Lehrke, Michael; Moellmann, Julia; Speer, Thimoteus; Buhl, Eva M; Hoogenboezem, Remco; Boor, Peter; Jansen, Jitske; Knopp, Cordula; Kurth, Ingo; Smeets, Bart; Bindels, Eric; Reinders, Marlies E J; Baan, Carla; Gribnau, Joost; Hoorn, Ewout J; Steffens, Joachim; Huber, Tobias B; Costa, Ivan; Floege, Jürgen; Schneider, Rebekka K; Saez-Rodriguez, Julio; Freedman, Benjamin S; Kramann, Rafael Nature genetics 36303074 2022 Oct 27
Prime editing for precise and highly versatile genome manipulation. Chen, Peter J; Liu, David R Nature reviews. Genetics 36344749 2023 Mar
Evolution of an adenine base editor into a small, efficient cytosine base editor with low off-target activity. Neugebauer, Monica E; Hsu, Alvin; Arbab, Mandana; Krasnow, Nicholas A; McElroy, Amber N; Pandey, Smriti; Doman, Jordan L; Huang, Tony P; Raguram, Aditya; Banskota, Samagya; Newby, Gregory A; Tolar, Jakub; Osborn, Mark J; Liu, David R Nature biotechnology 36357719 2022 Nov 10
LPA disruption with AAV-CRISPR potently lowers plasma apo(a) in transgenic mouse model: A proof-of-concept study. Doerfler, Alexandria M; Park, So Hyun; Assini, Julia M; Youssef, Amer; Saxena, Lavanya; Yaseen, Adam B; De Giorgi, Marco; Chuecos, Marcel; Hurley, Ayrea E; Li, Ang; Marcovina, Santica M; Bao, Gang; Boffa, Michael B; Koschinsky, Marlys L; Lagor, William R Molecular therapy. Methods & clinical development 36381302 2022 Dec 08
Production and characterization of virus-free, CRISPR-CAR T cells capable of inducing solid tumor regression. Mueller, Katherine P; Piscopo, Nicole J; Forsberg, Matthew H; Saraspe, Louise A; Das, Amritava; Russell, Brittany; Smerchansky, Madeline; Cappabianca, Dan; Shi, Lei; Shankar, Keerthana; Sarko, Lauren; Khajanchi, Namita; La Vonne Denne, Nina; Ramamurthy, A Journal for immunotherapy of cancer 36382633 2022 Sep
ABE8e adenine base editor precisely and efficiently corrects a recurrent COL7A1 nonsense mutation. Sheriff, Adam; Guri, Ina; Zebrowska, Paulina; Llopis-Hernandez, Virginia; Brooks, Imogen R; Tekkela, Stavroula; Subramaniam, Kavita; Gebrezgabher, Ruta; Naso, Gaetano; Petrova, Anastasia; Balon, Katarzyna; Onoufriadis, Alexandros; Kujawa, Dorota; Kotulska, Martyna; Newby, Gregory; Łaczmański, Łukasz; Liu, David R; McGrath, John A; Jacków, Joanna Scientific reports 36385635 2022 Nov 16
Compact zinc finger base editors that edit mitochondrial or nuclear DNA in vitro and in vivo. Willis, Julian C W; Silva-Pinheiro, Pedro; Widdup, Lily; Minczuk, Michal; Liu, David R Nature communications 36418298 2022 Nov 23
Diverse virus-encoded CRISPR-Cas systems include streamlined genome editors. Al-Shayeb, Basem; Skopintsev, Petr; Soczek, Katarzyna M; Stahl, Elizabeth C; Li, Zheng; Groover, Evan; Smock, Dylan; Eggers, Amy R; Pausch, Patrick; Cress, Brady F; Huang, Carolyn J; Staskawicz, Brian; Savage, David F; Jacobsen, Steven E; Banfield, Jillia Cell 36423580 2022 Nov 23
Substituting racemic ionizable lipids with stereopure ionizable lipids can increase mRNA delivery. Da Silva Sanchez, Alejandro J; Zhao, Kun; Huayamares, Sebastian G; Hatit, Marine Z C; Lokugamage, Melissa P; Loughrey, David; Dobrowolski, Curtis; Wang, Shuaishuai; Kim, Hyejin; Paunovska, Kalina; Kuzminich, Yanina; Dahlman, James E Journal of controlled release : official journal of the Controlled Release Society 36423872 2022 Nov 30
Drag-and-drop genome insertion of large sequences without double-strand DNA cleavage using CRISPR-directed integrases. Yarnall, Matthew T N; Ioannidi, Eleonora I; Schmitt-Ulms, Cian; Krajeski, Rohan N; Lim, Justin; Villiger, Lukas; Zhou, Wenyuan; Jiang, Kaiyi; Garushyants, Sofya K; Roberts, Nathaniel; Zhang, Liyang; Vakulskas, Christopher A; Walker 2nd, John A; Kadina, Anastasia P; Zepeda, Adrianna E; Holden, Kevin; Ma, Hong; Xie, Jun; Gao, Guangping; Foquet, Lander; Bial, Greg; Donnelly, Sara K; Miyata, Yoshinari; Radiloff, Daniel R; Henderson, Jordana M; Ujita, Andrew; Abudayyeh, Omar O; Gootenberg, Jonathan S Nature biotechnology 36424489 2023 Apr
Cyclin G1 induces maladaptive proximal tubule cell dedifferentiation and renal fibrosis through CDK5 activation. Taguchi, Kensei; Elias, Bertha C; Sugahara, Sho; Sant, Snehal; Freedman, Benjamin S; Waikar, Sushrut S; Pozzi, Ambra; Zent, Roy; Harris, Raymond C; Parikh, Samir M; Brooks, Craig R The Journal of clinical investigation 36453545 2022 Dec 01
Reframing the perception of outliers and negative data in translational research. Emborg, Marina E Brain research bulletin 36464129 2023 Jan
An enhancer-based gene-therapy strategy for spatiotemporal control of cargoes during tissue repair. Yan, Ruorong; Cigliola, Valentina; Oonk, Kelsey A; Petrover, Zachary; DeLuca, Sophia; Wolfson, David W; Vekstein, Andrew; Mendiola, Michelle A; Devlin, Garth; Bishawi, Muath; Gemberling, Matthew P; Sinha, Tanvi; Sargent, Michelle A; York, Allen J; Shakked, Avraham; DeBenedittis, Paige; Wendell, David C; Ou, Jianhong; Kang, Junsu; Goldman, Joseph A; Baht, Gurpreet S; Karra, Ravi; Williams, Adam R; Bowles, Dawn E; Asokan, Aravind; Tzahor, Eldad; Gersbach, Charles A; Molkentin, Jeffery D; Bursac, Nenad; Black, Brian L; Poss, Kenneth D Cell stem cell 36516837 2023 Jan 05
Efficient in vivo neuronal genome editing in the mouse brain using nanocapsules containing CRISPR-Cas9 ribonucleoproteins. Metzger, Jeanette M; Wang, Yuyuan; Neuman, Samuel S; Snow, Kathy J; Murray, Stephen A; Lutz, Cathleen M; Bondarenko, Viktoriya; Felton, Jesi; Gimse, Kirstan; Xie, Ruosen; Li, Dongdong; Zhao, Yi; Flowers, Matthew T; Simmons, Heather A; Roy, Subhojit; Saha, Krishanu; Levine, Jon E; Emborg, Marina E; Gong, Shaoqin Biomaterials 36527789 2022 Dec 13
Emerging immunomodulatory strategies for cell therapeutics. Chua, Corrine Ying Xuan; Jiang, Allen Yujie; Eufrásio-da-Silva, Tatiane; Dolatshahi-Pirouz, Alireza; Langer, Robert; Orive, Gorka; Grattoni, Alessandro Trends in biotechnology 36549959 2023 Mar
Glucose absorption drives cystogenesis in a human organoid-on-chip model of polycystic kidney disease. Li, Sienna R; Gulieva, Ramila E; Helms, Louisa; Cruz, Nelly M; Vincent, Thomas; Fu, Hongxia; Himmelfarb, Jonathan; Freedman, Benjamin S Nature communications 36564419 2022 Dec 23
Rigid respiration: fulminant pulmonary fibrosis after COVID-19. Meyerholz, David K EBioMedicine 36580850 2023 Jan
In vivo editing of the pan-endothelium by immunity evading simian adenoviral vector. Lorincz, Reka; Alvarez, Aluet Borrego; Walkey, Christopher J; Mendonça, Samir A; Lu, Zhi Hong; Martinez, Alexa E; Ljungberg, Cecilia; Heaney, Jason D; Lagor, William R; Curiel, David T Biomedicine & pharmacotherapy = Biomedecine & pharmacotherapie 36587560 2023 Feb
In Vivo Cellular Magnetic Imaging: Labeled vs. Unlabeled Cells. Bulte, Jeff W M; Wang, Chao; Shakeri-Zadeh, Ali Advanced functional materials 36589903 2022 Dec 09
Accounting for diversity in the design of CRISPR-based therapeutic genome editing. Saha, Krishanu Nature genetics 36593304 2023 Jan
Synthetic Biology Design as a Paradigm Shift toward Manufacturing Affordable Adeno-Associated Virus Gene Therapies. Collins, Logan Thrasher; Ponnazhagan, Selvarangan; Curiel, David T ACS synthetic biology 36627108 2023 Jan 20
Phenylboronic Acid-Functionalized Polyplexes Tailored to Oral CRISPR Delivery. Yoshinaga, Naoto; Zhou, Joyce K; Xu, Cong; Quek, Chai Hoon; Zhu, Yuefei; Tang, Ding; Hung, Lin Yung; Najjar, Sarah A; Shiu, Chin Ying Angela; Margolis, Kara Gross; Lao, Yeh-Hsing; Leong, Kam W Nano letters 36648291 2023 Feb 08
Clinical progress in genome-editing technology and in vivo delivery techniques. Khirallah, Jennifer; Eimbinder, Maximilan; Li, Yamin; Xu, Qiaobing Trends in genetics : TIG 36669950 2023 Jan 18
Precise transcript targeting by CRISPR-Csm complexes. Colognori, David; Trinidad, Marena; Doudna, Jennifer A Nature biotechnology 36690762 2023 Sep
The Transcriptional Response to Lung-Targeting Lipid Nanoparticles in Vivo. Radmand, Afsane; Lokugamage, Melissa P; Kim, Hyejin; Dobrowolski, Curtis; Zenhausern, Ryan; Loughrey, David; Huayamares, Sebastian G; Hatit, Marine Z C; Ni, Huanzhen; Del Cid, Ada; Da Silva Sanchez, Alejandro J; Paunovska, Kalina; Schrader Echeverri, Elisa; Shajii, Aram; Peck, Hannah; Santangelo, Philip J; Dahlman, James E Nano letters 36701517 2023 Feb 08
Base editing as a genetic treatment for spinal muscular atrophy. Alves, Christiano R R; Ha, Leillani L; Yaworski, Rebecca; Lazzarotto, Cicera R; Christie, Kathleen A; Reilly, Aoife; Beauvais, Ariane; Doll, Roman M; de la Cruz, Demitri; Maguire, Casey A; Swoboda, Kathryn J; Tsai, Shengdar Q; Kothary, Rashmi; Kleinstiver, Benjamin P bioRxiv : the preprint server for biology 36711797 2023 Jan 21
Focused ultrasound-mediated brain genome editing. Leong, Kam; Lao, Yeh-Hsing; Ji, Robin; Zhou, Joyce; Snow, Kathy; Kwon, Nancy; Saville, Ethan; He, Siyu; Chauhan, Shradha; Chi, Chun-Wei; Datta, Malika; Zhang, Hairong; Quek, Chai Hoon; Cai, Sarah; Li, Mingqiang; Gaitan, Yaned; Bechtel, Lawrence; Wu, Shih-Ying; Lutz, Cathleen; Tomer, Raju; Murray, Stephen; Chavez, Alejandro; Konofagou, Elisa Research square 36712096 2023 Jan 20
Functional maturation of kidney organoid tubules: Piezo1-mediated Ca2+ signaling. Carrisoza-Gaytán, Rolando; Kroll, Katharina T; Hiratsuka, Ken; Gupta, Navin R; Morizane, Ryuji; Lewis, Jennifer A; Satlin, Lisa M American journal of physiology. Cell physiology 36745528 2023 Feb 06
Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in mice. Reichart, Daniel; Newby, Gregory A; Wakimoto, Hiroko; Lun, Mingyue; Gorham, Joshua M; Curran, Justin J; Raguram, Aditya; DeLaughter, Daniel M; Conner, David A; Marsiglia, Júlia D C; Kohli, Sajeev; Chmatal, Lukas; Page, David C; Zabaleta, Nerea; Vandenberghe, Luk; Liu, David R; Seidman, Jonathan G; Seidman, Christine Nature medicine 36797483 2023 Feb
In vivo HSC prime editing rescues sickle cell disease in a mouse model. Li, Chang; Georgakopoulou, Aphrodite; Newby, Gregory A; Chen, Peter J; Everette, Kelcee A; Paschoudi, Kiriaki; Vlachaki, Efthymia; Gil, Sucheol; Anderson, Anna K; Koob, Theodore; Huang, Lishan; Wang, Hongjie; Kiem, Hans-Peter; Liu, David R; Yannaki, Evangelia; Lieber, André Blood 36800642 2023 Apr 27
Shuttle Peptide Delivers Base Editor RNPs to Rhesus Monkey Airway Epithelial Cells In Vivo. Kulhankova, Katarina; Traore, Soumba; Cheng, Xue; Benk-Fortin, Hadrien; Hallée, Stéphanie; Harvey, Mario; Roberge, Joannie; Couture, Frédéric; Gross, Thomas; Newby, Gregory; Liu, David; Tarantal, Alice; Guay, David; McCray, Paul Research square 36824928 2023 Feb 17
In utero delivery of mRNA to the heart, diaphragm and muscle with lipid nanoparticles. Gao, Kewa; Li, Jie; Song, Hengyue; Han, Hesong; Wang, Yongheng; Yin, Boyan; Farmer, Diana L; Murthy, Niren; Wang, Aijun Bioactive materials 36844366 2023 Jul
A parathyroid hormone/salt-inducible kinase signaling axis controls renal vitamin D activation and organismal calcium homeostasis. Yoon, Sung-Hee; Meyer, Mark B; Arevalo Rivas, Carlos; Tekguc, Murat; Zhang, Chengcheng; Wang, Jialiang S; Castro Andrade, Christian D; Strauss, Katelyn E; Sato, Tadatoshi; Benkusky, Nancy; Lee, Seong Min; Berdeaux, Rebecca; Foretz, Marc; Sundberg, Thomas B; Xavier, Ramnik J; Adelmann, Charles H; Brooks, Daniel J; Anselmo, Anthony; Sadreyev, Ruslan I; Rosales, Ivy A; Fisher, David E; Gupta, Navin; Morizane, Ryuji; Greka, Anna; Pike, J Wesley; Mannstadt, Michael; Wein, Marc N The Journal of clinical investigation 36862513 2023 Mar 02
Nanoparticle stereochemistry-dependent endocytic processing improves in vivo mRNA delivery. Hatit, Marine Z C; Dobrowolski, Curtis N; Lokugamage, Melissa P; Loughrey, David; Ni, Huanzhen; Zurla, Chiara; Da Silva Sanchez, Alejandro J; Radmand, Afsane; Huayamares, Sebastian G; Zenhausern, Ryan; Paunovska, Kalina; Peck, Hannah E; Kim, Jinwhan; Sato, Manaka; Feldman, Jacob I; Rivera, Michael-Alexander; Cristian, Ana; Kim, YongTae; Santangelo, Philip J; Dahlman, James E Nature chemistry 36864143 2023 Apr
The history, use, and challenges of therapeutic somatic cell and germline gene editing. Ryu, Junghyun; Adashi, Eli Y; Hennebold, Jon D Fertility and sterility 36878350 2023 Sep
Human T cell generation is restored in CD3δ severe combined immunodeficiency through adenine base editing. McAuley, Grace E; Yiu, Gloria; Chang, Patrick C; Newby, Gregory A; Campo-Fernandez, Beatriz; Fitz-Gibbon, Sorel T; Wu, Xiaomeng; Kang, Sung-Hae L; Garibay, Amber; Butler, Jeffrey; Christian, Valentina; Wong, Ryan L; Everette, Kelcee A; Azzun, Anthony; Gelfer, Hila; Seet, Christopher S; Narendran, Aru; Murguia-Favela, Luis; Romero, Zulema; Wright, Nicola; Liu, David R; Crooks, Gay M; Kohn, Donald B Cell 36944331 2023 Mar 30
Mitochondrial Base Editing: Recent Advances towards Therapeutic Opportunities. Kar, Bibekananda; Castillo, Santiago R; Sabharwal, Ankit; Clark, Karl J; Ekker, Stephen C International journal of molecular sciences 36982871 2023 Mar 18
Self-delivering CRISPR RNAs for AAV Co-delivery and Genome Editing in vivo. Zhang, Han; Kelly, Karen; Lee, Jonathan; Echeverria, Dimas; Cooper, David; Panwala, Rebecca; Chen, Zexiang; Gaston, Nicholas; Newby, Gregory A; Xie, Jun; Liu, David R; Gao, Guangping; Wolfe, Scot A; Khvorova, Anastasia; Watts, Jonathan K; Sontheimer, Erik J bioRxiv : the preprint server for biology 36993169 2023 Mar 23
Mitigation of chromosome loss in clinical CRISPR-Cas9-engineered T cells. Tsuchida, Connor A; Brandes, Nadav; Bueno, Raymund; Trinidad, Marena; Mazumder, Thomas; Yu, Bingfei; Hwang, Byungjin; Chang, Christopher; Liu, Jamin; Sun, Yang; Hopkins, Caitlin R; Parker, Kevin R; Qi, Yanyan; Satpathy, Ansuman T; Stadtmauer, Edward A; Cate, Jamie H D; Eyquem, Justin; Fraietta, Joseph A; June, Carl H; Chang, Howard Y; Ye, Chun Jimmie; Doudna, Jennifer A bioRxiv : the preprint server for biology 36993359 2023 Mar 22
Base editing rescue of spinal muscular atrophy in cells and in mice. Arbab, Mandana; Matuszek, Zaneta; Kray, Kaitlyn M; Du, Ailing; Newby, Gregory A; Blatnik, Anton J; Raguram, Aditya; Richter, Michelle F; Zhao, Kevin T; Levy, Jonathan M; Shen, Max W; Arnold, W David; Wang, Dan; Xie, Jun; Gao, Guangping; Burghes, Arthur H M; Liu, David R Science (New York, N.Y.) 36996170 2023 Apr 21
Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing. Li, Bowen; Manan, Rajith Singh; Liang, Shun-Qing; Gordon, Akiva; Jiang, Allen; Varley, Andrew; Gao, Guangping; Langer, Robert; Xue, Wen; Anderson, Daniel Nature biotechnology 36997680 2023 Oct
Rescue of GM3 synthase deficiency by spatially controlled, rAAV-mediated ST3GAL5 delivery. Yang, Huiya; Brown Jr, Robert H; Wang, Dan; Strauss, Kevin A; Gao, Guangping JCI insight 37014712 2023 May 08
Adeno-Associated virus 8 delivers an immunomodulatory peptide to mouse liver more efficiently than to rat liver. Wang, Yuqing; Hurley, Ayrea; De Giorgi, Marco; Tanner, Mark R; Hu, Rong-Chi; Pennington, Michael W; Lagor, William R; Beeton, Christine PloS one 37040361 2023
CaMKIIδ gene editing- A base hit for the heart. Walkey, Christopher J; Lagor, William R The journal of cardiovascular aging 37065676 2023
Reprogramming by drug-like molecules leads to regeneration of cochlear hair cell-like cells in adult mice. Quan, Yi-Zhou; Wei, Wei; Ergin, Volkan; Rameshbabu, Arun Prabhu; Huang, Mingqian; Tian, Chunjie; Saladi, Srinivas Vinod; Indzhykulian, Artur A; Chen, Zheng-Yi Proceedings of the National Academy of Sciences of the United States of America 37068229 2023 Apr 25
Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice. Everette, Kelcee A; Newby, Gregory A; Levine, Rachel M; Mayberry, Kalin; Jang, Yoonjeong; Mayuranathan, Thiyagaraj; Nimmagadda, Nikitha; Dempsey, Erin; Li, Yichao; Bhoopalan, Senthil Velan; Liu, Xiong; Davis, Jessie R; Nelson, Andrew T; Chen, Peter J; Sousa, Alexander A; Cheng, Yong; Tisdale, John F; Weiss, Mitchell J; Yen, Jonathan S; Liu, David R Nature biomedical engineering 37069266 2023 May
Strategies for Improving Vascularization in Kidney Organoids: A Review of Current Trends. Konoe, Ran; Morizane, Ryuji Biology 37106704 2023 Mar 26
HIV-Differentiated Metabolite N-Acetyl-L-Alanine Dysregulates Human Natural Killer Cell Responses to Mycobacterium tuberculosis Infection. Yang, Baojun; Mukherjee, Tanmoy; Radhakrishnan, Rajesh; Paidipally, Padmaja; Ansari, Danish; John, Sahana; Vankayalapati, Ramakrishna; Tripathi, Deepak; Yi, Guohua International journal of molecular sciences 37108430 2023 Apr 14
Massively parallel base editing to map variant effects in human hematopoiesis. Martin-Rufino, Jorge D; Castano, Nicole; Pang, Michael; Grody, Emanuelle I; Joubran, Samantha; Caulier, Alexis; Wahlster, Lara; Li, Tongqing; Qiu, Xiaojie; Riera-Escandell, Anna Maria; Newby, Gregory A; Al'Khafaji, Aziz; Chaudhary, Santosh; Black, Susan; Weng, Chen; Munson, Glen; Liu, David R; Wlodarski, Marcin W; Sims, Kacie; Oakley, Jamie H; Fasano, Ross M; Xavier, Ramnik J; Lander, Eric S; Klein, Daryl E; Sankaran, Vijay G Cell 37137305 2023 May 25
Efficient prime editing in mouse brain, liver and heart with dual AAVs. Davis, Jessie R; Banskota, Samagya; Levy, Jonathan M; Newby, Gregory A; Wang, Xiao; Anzalone, Andrew V; Nelson, Andrew T; Chen, Peter J; Hennes, Andrew D; An, Meirui; Roh, Heejin; Randolph, Peyton B; Musunuru, Kiran; Liu, David R Nature biotechnology 37142705 2023 May 04
Peptide-mediated delivery of CRISPR enzymes for the efficient editing of primary human lymphocytes. Foss, Dana V; Muldoon, Joseph J; Nguyen, David N; Carr, Daniel; Sahu, Srishti U; Hunsinger, John M; Wyman, Stacia K; Krishnappa, Netravathi; Mendonsa, Rima; Schanzer, Elaine V; Shy, Brian R; Vykunta, Vivasvan S; Allain, Vincent; Li, Zhongmei; Marson, Alexander; Eyquem, Justin; Wilson, Ross C Nature biomedical engineering 37147433 2023 May
Renewal of oligodendrocyte lineage reverses dysmyelination and CNS neurodegeneration through corrected N-acetylaspartate metabolism. Lotun, Anoushka; Li, Danning; Xu, Hongxia; Su, Qin; Tuncer, Serafettin; Sanmiguel, Julio; Mooney, Morgan; Baer, Christina E; Ulbrich, Russell; Eyles, Stephen J; Strittmatter, Lara; Hayward, Lawrence J; Gessler, Dominic J; Gao, Guangping Progress in neurobiology 37149081 2023 Jul
A Multifunctional and Highly Adaptable Reporter System for CRISPR/Cas Editing. Wettengel, Jochen M; Hansen-Palmus, Lea; Yusova, Sofiya; Rust, Lauren; Biswas, Sreya; Carson, Julien; Ryu, Junghyun; Bimber, Benjamin N; Hennebold, Jon D; Burwitz, Benjamin J International journal of molecular sciences 37175977 2023 May 05
Genetic background in the rat affects endocrine and metabolic outcomes of bisphenol F exposure. Wagner, Valerie A; Holl, Katie L; Clark, Karen C; Reho, John J; Dwinell, Melinda R; Lehmler, Hans-Joachim; Raff, Hershel; Grobe, Justin L; Kwitek, Anne E Toxicological sciences : an official journal of the Society of Toxicology 37191987 2023 Jun 28
Orthogonal CRISPR screens to identify transcriptional and epigenetic regulators of human CD8 T cell function. McCutcheon, Sean R; Swartz, Adam M; Brown, Michael C; Barrera, Alejandro; Amador, Christian McRoberts; Siklenka, Keith; Humayun, Lucas; Isaacs, James M; Reddy, Timothy E; Nair, Smita; Antonia, Scott; Gersbach, Charles A bioRxiv : the preprint server for biology 37205457 2023 May 01
Rescue of auditory function by a single administration of AAV-TMPRSS3 gene therapy in aged mice of human recessive deafness DFNB8. Du, Wan; Ergin, Volkan; Loeb, Corena; Huang, Mingqian; Silver, Stewart; Armstrong, Ariel Miura; Huang, Zaohua; Gurumurthy, Channabasavaiah B; Staecker, Hinrich; Liu, Xuezhong; Chen, Zheng-Yi Molecular therapy : the journal of the American Society of Gene Therapy 37244253 2023 May 22
Reducing the inherent auto-inhibitory interaction within the pegRNA enhances prime editing efficiency. Ponnienselvan, Karthikeyan; Liu, Pengpeng; Nyalile, Thomas; Oikemus, Sarah; Maitland, Stacy A; Lawson, Nathan D; Luban, Jeremy; Wolfe, Scot A Nucleic acids research 37246708 2023 Jul 21
Liver directed adeno-associated viral vectors to treat metabolic disease. Chuecos, Marcel A; Lagor, William R Journal of inherited metabolic disease 37254440 2023 May 30
Template-jumping prime editing enables large insertion and exon rewriting in vivo. Zheng, Chunwei; Liu, Bin; Dong, Xiaolong; Gaston, Nicholas; Sontheimer, Erik J; Xue, Wen Nature communications 37291100 2023 Jun 08
Rapid and definitive treatment of phenylketonuria in variant-humanized mice with corrective editing. Brooks, Dominique L; Carrasco, Manuel J; Qu, Ping; Peranteau, William H; Ahrens-Nicklas, Rebecca C; Musunuru, Kiran; Alameh, Mohamad-Gabriel; Wang, Xiao Nature communications 37301931 2023 Jun 10
Mercapto-pyrimidines are reversible covalent inhibitors of the papain-like protease (PLpro) and inhibit SARS-CoV-2 (SCoV-2) replication. Bajaj, Teena; Wehri, Eddie; Suryawanshi, Rahul K; King, Elizabeth; Pardeshi, Kundan Singh; Behrouzi, Kamyar; Khodabakhshi, Zahra; Schulze-Gahmen, Ursula; Kumar, G Renuka; Mofrad, Mohammad R K; Nomura, Daniel K; Ott, Melanie; Schaletzky, Julia; Murthy, Niren RSC advances 37312993 2023 Jun 09
Scalable projected Light Sheet Microscopy for high-resolution imaging of large samples. Chen, Yannan; Gong, Cheng; Chauhan, Shradha; De La Cruz, Estanislao Daniel; Datta, Malika S; Tomer, Raju bioRxiv : the preprint server for biology 37333196 2023 Jun 05
Querying Recombination Junctions of Replication-Competent Adeno-Associated Viruses in Gene Therapy Vector Preparations with Single Molecule, Real-Time Sequencing. Yip, Mitchell; Chen, Jing; Zhi, Yan; Tran, Ngoc Tam; Namkung, Suk; Pastor, Eric; Gao, Guangping; Tai, Phillip W L Viruses 37376529 2023 May 24
Utilizing directed evolution to interrogate and optimize CRISPR/Cas guide RNA scaffolds. Bush, Korie; Corsi, Giulia I; Yan, Amy C; Haynes, Keith; Layzer, Juliana M; Zhou, Jonathan H; Llanga, Telmo; Gorodkin, Jan; Sullenger, Bruce A Cell chemical biology 37390831 2023 Aug 17
Potent and uniform fetal hemoglobin induction via base editing. Mayuranathan, Thiyagaraj; Newby, Gregory A; Feng, Ruopeng; Yao, Yu; Mayberry, Kalin D; Lazzarotto, Cicera R; Li, Yichao; Levine, Rachel M; Nimmagadda, Nikitha; Dempsey, Erin; Kang, Guolian; Porter, Shaina N; Doerfler, Phillip A; Zhang, Jingjing; Jang, Yoonjeong; Chen, Jingjing; Bell, Henry W; Crossley, Merlin; Bhoopalan, Senthil Velan; Sharma, Akshay; Tisdale, John F; Pruett-Miller, Shondra M; Cheng, Yong; Tsai, Shengdar Q; Liu, David R; Weiss, Mitchell J; Yen, Jonathan S Nature genetics 37400614 2023 Jul
Rational nanoparticle design: Optimization using insights from experiments and mathematical models. Richfield, Owen; Piotrowski-Daspit, Alexandra S; Shin, Kwangsoo; Saltzman, W Mark Journal of controlled release : official journal of the Controlled Release Society 37442201 2023 Aug
Adenoviral vectors infect B lymphocytes in vivo. Rice-Boucher, Paul J; Mendonça, Samir Andrade; Alvarez, Aluet Borrego; Sturtz, Alexandria J; Lorincz, Reka; Dmitriev, Igor P; Kashentseva, Elena A; Lu, Zhi Hong; Romano, Rosa; Selby, Mark; Pingale, Kunal; Curiel, David T Molecular therapy : the journal of the American Society of Gene Therapy 37452494 2023 Sep 06
Targeting AAV vectors to the central nervous system by engineering capsid-receptor interactions that enable crossing of the blood-brain barrier. Huang, Qin; Chen, Albert T; Chan, Ken Y; Sorensen, Hikari; Barry, Andrew J; Azari, Bahar; Zheng, Qingxia; Beddow, Thomas; Zhao, Binhui; Tobey, Isabelle G; Moncada-Reid, Cynthia; Eid, Fatma-Elzahraa; Walkey, Christopher J; Ljungberg, M Cecilia; Lagor, William R; Heaney, Jason D; Chan, Yujia A; Deverman, Benjamin E PLoS biology 37467291 2023 Jul
Gene editing therapeutics based on mRNA delivery. Popovitz, Juliana; Sharma, Rohit; Hoshyar, Reyhane; Soo Kim, Beob; Murthy, Niren; Lee, Kunwoo Advanced drug delivery reviews 37516409 2023 Sep
Individually tailored spatial-spectral pulsed CEST MRI for ratiometric mapping of myocardial energetic species at 3T. Ayala, Cindy; Luo, Huiwen; Godines, Kevin; Alghuraibawi, Wissam; Ahn, Sinyeob; Rehwald, Wolfgang; Grissom, William A; Vandsburger, Moriel H Magnetic resonance in medicine 37526176 2023 Dec
In vivo expansion of gene-targeted hepatocytes through transient inhibition of an essential gene. De Giorgi, Marco; Park, So Hyun; Castoreno, Adam; Cao, Mingming; Hurley, Ayrea; Saxena, Lavanya; Chuecos, Marcel A; Walkey, Christopher J; Doerfler, Alexandria M; Furgurson, Mia N; Ljungberg, M Cecilia; Patel, Kalyani R; Hyde, Sarah; Chickering, Tyler; Lefebvre, Stephanie; Wassarman, Kelly; Miller, Patrick; Qin, June; Schlegel, Mark K; Zlatev, Ivan; Li, Rich Gang; Kim, Jong; Martin, James F; Bissig, Karl-Dimiter; Jadhav, Vasant; Bao, Gang; Lagor, William R bioRxiv : the preprint server for biology 37546995 2023 Jul 29
Protospacer modification improves base editing of a canonical splice site variant and recovery of CFTR function in human airway epithelial cells. Joynt, Anya T; Kavanagh, Erin W; Newby, Gregory A; Mitchell, Shakela; Eastman, Alice C; Paul, Kathleen C; Bowling, Alyssa D; Osorio, Derek L; Merlo, Christian A; Patel, Shivani U; Raraigh, Karen S; Liu, David R; Sharma, Neeraj; Cutting, Garry R Molecular therapy. Nucleic acids 37547293 2023 Sep 12
Nonviral base editing of KCNJ13 mutation preserves vision in a model of inherited retinal channelopathy. Kabra, Meha; Shahi, Pawan K; Wang, Yuyuan; Sinha, Divya; Spillane, Allison; Newby, Gregory A; Saxena, Shivani; Tong, Yao; Chang, Yu; Abdeen, Amr A; Edwards, Kimberly L; Theisen, Cole O; Liu, David R; Gamm, David M; Gong, Shaoqin; Saha, Krishanu; Pattnaik, Bikash R The Journal of clinical investigation 37561581 2023 Oct 02
Focused ultrasound-mediated brain genome editing. Lao, Yeh-Hsing; Ji, Robin; Zhou, Joyce K; Snow, Kathy J; Kwon, Nancy; Saville, Ethan; He, Siyu; Chauhan, Shradha; Chi, Chun-Wei; Datta, Malika S; Zhang, Hairong; Quek, Chai Hoon; Cai, S Sarah; Li, Mingqiang; Gaitan, Yaned; Bechtel, Lawrence; Wu, Shih-Ying; Lutz, Cathleen M; Tomer, Raju; Murray, Stephen A; Chavez, Alejandro; Konofagou, Elisa E; Leong, Kam W Proceedings of the National Academy of Sciences of the United States of America 37579143 2023 Aug 22
Nonviral In Vivo Delivery of CRISPR-Cas9 Using Protein-Agnostic, High-Loading Porous Silicon and Polymer Nanoparticles. Fletcher, R Brock; Stokes, Larry D; Kelly 3rd, Isom B; Henderson, Katelyn M; Vallecillo-Viejo, Isabel C; Colazo, Juan M; Wong, Benjamin V; Yu, Fang; d'Arcy, Richard; Struthers, Morgan N; Evans, Brian C; Ayers, Jacob; Castanon, Matthew; Weirich, Michael J; Reilly, Sarah K; Patel, Shrusti S; Ivanova, Yoanna I; Silvera Batista, Carlos A; Weiss, Sharon M; Gersbach, Charles A; Brunger, Jonathan M; Duvall, Craig L ACS nano 37582231 2023 Sep 12
Treatment of monogenic and digenic dominant genetic hearing loss by CRISPR-Cas9 ribonucleoprotein delivery in vivo. Tao, Yong; Lamas, Veronica; Du, Wan; Zhu, Wenliang; Li, Yiran; Whittaker, Madelynn N; Zuris, John A; Thompson, David B; Rameshbabu, Arun Prabhu; Shu, Yilai; Gao, Xue; Hu, Johnny H; Pei, Charles; Kong, Wei-Jia; Liu, Xuezhong; Wu, Hao; Kleinstiver, Benjamin P; Liu, David R; Chen, Zheng-Yi Nature communications 37582836 2023 Aug 15
Polymer nanoparticles deliver mRNA to the lung for mucosal vaccination. Suberi, Alexandra; Grun, Molly K; Mao, Tianyang; Israelow, Benjamin; Reschke, Melanie; Grundler, Julian; Akhtar, Laiba; Lee, Teresa; Shin, Kwangsoo; Piotrowski-Daspit, Alexandra S; Homer, Robert J; Iwasaki, Akiko; Suh, Hee-Won; Saltzman, W Mark Science translational medicine 37585505 2023 Aug 16
Phage-assisted evolution and protein engineering yield compact, efficient prime editors. Doman, Jordan L; Pandey, Smriti; Neugebauer, Monica E; An, Meirui; Davis, Jessie R; Randolph, Peyton B; McElroy, Amber; Gao, Xin D; Raguram, Aditya; Richter, Michelle F; Everette, Kelcee A; Banskota, Samagya; Tian, Kathryn; Tao, Y Allen; Tolar, Jakub; Osborn, Mark J; Liu, David R Cell 37657419 2023 Aug 31
Trichostatin A for Efficient CRISPR-Cas9 Gene Editing of Human Pluripotent Stem Cells. Molugu, Kaivalya; Khajanchi, Namita; Lazzarotto, Cicera R; Tsai, Shengdar Q; Saha, Krishanu The CRISPR journal 37676985 2023 Oct
Lipid Nanoparticles Deliver mRNA to the Brain after an Intracerebral Injection. Tuma, Jan; Chen, Yu-Ju; Collins, Michael G; Paul, Abhik; Li, Jie; Han, Hesong; Sharma, Rohit; Murthy, Niren; Lee, Hye Young Biochemistry 37729550 2023 Dec 19
Structure-guided AAV capsid evolution strategies for enhanced CNS gene delivery. Gonzalez, Trevor J; Mitchell-Dick, Aaron; Blondel, Leo O; Fanous, Marco M; Hull, Joshua A; Oh, Daniel K; Moller-Tank, Sven; Castellanos Rivera, Ruth M; Piedrahita, Jorge A; Asokan, Aravind Nature protocols 37735235 2023 Nov
A brown fat-enriched adipokine, ASRA, is a leptin receptor antagonist that stimulates appetite. Huang, Lei; Liu, Pengpeng; Du, Yong; Pan, Dongning; Lee, Alexandra; Wolfe, Scot A; Wang, Yong-Xu bioRxiv : the preprint server for biology 37745491 2023 Sep 12
Navigating the kidney organoid: insights into assessment and enhancement of nephron function. Tabibzadeh, Nahid; Satlin, Lisa M; Jain, Sanjay; Morizane, Ryuji American journal of physiology. Renal physiology 37767571 2023 Dec 01
Assigning functionality to cysteines by base editing of cancer dependency genes. Li, Haoxin; Ma, Tiantai; Remsberg, Jarrett R; Won, Sang Joon; DeMeester, Kristen E; Njomen, Evert; Ogasawara, Daisuke; Zhao, Kevin T; Huang, Tony P; Lu, Bingwen; Simon, Gabriel M; Melillo, Bruno; Schreiber, Stuart L; Lykke-Andersen, Jens; Liu, David R; Cravatt, Benjamin F Nature chemical biology 37783940 2023 Nov
Mitigation of chromosome loss in clinical CRISPR-Cas9-engineered T cells. Tsuchida, Connor A; Brandes, Nadav; Bueno, Raymund; Trinidad, Marena; Mazumder, Thomas; Yu, Bingfei; Hwang, Byungjin; Chang, Christopher; Liu, Jamin; Sun, Yang; Hopkins, Caitlin R; Parker, Kevin R; Qi, Yanyan; Hofman, Laura; Satpathy, Ansuman T; Stadtmauer, Edward A; Cate, Jamie H D; Eyquem, Justin; Fraietta, Joseph A; June, Carl H; Chang, Howard Y; Ye, Chun Jimmie; Doudna, Jennifer A Cell 37794590 2023 Oct 12
Redirecting AAV vectors to extrahepatic tissues. Asokan, Aravind; Shen, Shen Molecular therapy : the journal of the American Society of Gene Therapy 37805712 2023 Dec 06
Avian influenza A viruses exhibit plasticity in sialylglycoconjugate receptor usage in human lung cells. Liang, Chieh-Yu; Huang, Iris; Han, Julianna; Sownthirarajan, Boopathi; Kulhankova, Katarina; Murray, Nathan B; Taherzadeh, Mehrnoush; Archer-Hartmann, Stephanie; Pepi, Lauren; Manivasagam, Senthamizharasi; Plung, Jesse; Sturtz, Miranda; Yu, Yolanda; Vogel, Olivia A; Kandasamy, Matheswaran; Gourronc, Francoise A; Klingelhutz, Aloysius J; Choudhury, Biswa; Rong, Lijun; Perez, Jasmine T; Azadi, Parastoo; McCray Jr, Paul B; Neelamegham, Sriram; Manicassamy, Balaji Journal of virology 37843369 2023 Nov 30
PCSK9 activation promotes early atherosclerosis in a vascular microphysiological system. Lee, Jounghyun H; Shores, Kevin L; Breithaupt, Jason J; Lee, Caleb S; Fodera, Daniella M; Kwon, Jennifer B; Ettyreddy, Adarsh R; Myers, Kristin M; Evison, Benny J; Suchowerska, Alexandra K; Gersbach, Charles A; Leong, Kam W; Truskey, George A APL bioengineering 37854060 2023 Dec
The Power of the Heterogeneous Stock Rat Founder Strains in Modeling Metabolic Disease. Wagner, Valerie A; Holl, Katie L; Clark, Karen C; Reho, John J; Lehmler, Hans-Joachim; Wang, Kai; Grobe, Justin L; Dwinell, Melinda R; Raff, Hershel; Kwitek, Anne E Endocrinology 37882530 2023 Nov 02
CasPEDIA Database: a functional classification system for class 2 CRISPR-Cas enzymes. Adler, Benjamin A; Trinidad, Marena I; Bellieny-Rabelo, Daniel; Zhang, Elaine; Karp, Hannah M; Skopintsev, Petr; Thornton, Brittney W; Weissman, Rachel F; Yoon, Peter H; Chen, LinXing; Hessler, Tomas; Eggers, Amy R; Colognori, David; Boger, Ron; Doherty, Erin E; Tsuchida, Connor A; Tran, Ryan V; Hofman, Laura; Shi, Honglue; Wasko, Kevin M; Zhou, Zehan; Xia, Chenglong; Al-Shimary, Muntathar J; Patel, Jaymin R; Thomas, Vienna C J X; Pattali, Rithu; Kan, Matthew J; Vardapetyan, Anna; Yang, Alana; Lahiri, Arushi; Maxwell, Micaela F; Murdock, Andrew G; Ramit, Glenn C; Henderson, Hope R; Calvert, Roland W; Bamert, Rebecca S; Knott, Gavin J; Lapinaite, Audrone; Pausch, Patrick; Cofsky, Joshua C; Sontheimer, Erik J; Wiedenheft, Blake; Fineran, Peter C; Brouns, Stan J J; Sashital, Dipali G; Thomas, Brian C; Brown, Christopher T; Goltsman, Daniela S A; Barrangou, Rodolphe; Siksnys, Virginius; Banfield, Jillian F; Savage, David F; Doudna, Jennifer A Nucleic acids research 37889041 2024 Jan 05
DNA recognition and induced genome modification by a hydroxymethyl-γ tail-clamp peptide nucleic acid. Oyaghire, Stanley N; Quijano, Elias; Perera, J Dinithi R; Mandl, Hanna K; Saltzman, W Mark; Bahal, Raman; Glazer, Peter M Cell reports. Physical science 37920723 2023 Oct 18
Efficient in vivo prime editing corrects the most frequent phenylketonuria variant, associated with high unmet medical need. Brooks, Dominique L; Whittaker, Madelynn N; Qu, Ping; Musunuru, Kiran; Ahrens-Nicklas, Rebecca C; Wang, Xiao American journal of human genetics 37924808 2023 Dec 07
Building blocks for better biorepositories in Africa. Croxton, Talishiea; Jonathan, Emmanuel; Suleiman, Kareemah; Balogun, Olasinbo; Ozumba, Petronilla J; Aloyo, Sharley M; Nsubuga, Gideon; Kamulegeya, Rogers E; Newton, Lwanga; Mukisa, John; Kader, Mukthar; Damaneite, Vuyo; Nadoma, Sunji; Onyemata, Ezenwa James; Anzaku, Abbas Abel; Nasinghe, Emmanuel; Troyer, Jennifer; Joubert, Bonnie R; Beiswanger, Christine; Joloba, Moses L; Mayne, Elizabeth; Abimiku, Alash'le; H3Africa Biorepository PI Working Group Genome medicine 37932809 2023 Nov 06
Human red blood cells release microvesicles with distinct sizes and protein composition that alter neutrophil phagocytosis. de Oliveira Junior, Getulio Pereira; Welsh, Joshua A; Pinckney, Brandy; Palu, Cintia C; Lu, Shulin; Zimmerman, Alan; Barbosa, Raquel Hora; Sahu, Parul; Noshin, Maeesha; Gummuluru, Suryaram; Tigges, John; Jones, Jennifer Clare; Ivanov, Alexander R; Ghiran, Ionita C Journal of extracellular biology 37942280 2023 Nov
Transcriptional and epigenetic regulators of human CD8+ T cell function identified through orthogonal CRISPR screens. McCutcheon, Sean R; Swartz, Adam M; Brown, Michael C; Barrera, Alejandro; McRoberts Amador, Christian; Siklenka, Keith; Humayun, Lucas; Ter Weele, Maria A; Isaacs, James M; Reddy, Timothy E; Allen, Andrew S; Nair, Smita K; Antonia, Scott J; Gersbach, Charles A Nature genetics 37945901 2023 Dec
Eukaryotic RNA-guided endonucleases evolved from a unique clade of bacterial enzymes. Yoon, Peter H; Skopintsev, Petr; Shi, Honglue; Chen, LinXing; Adler, Benjamin A; Al-Shimary, Muntathar; Craig, Rory J; Loi, Kenneth J; DeTurk, Evan C; Li, Zheng; Amerasekera, Jasmine; Trinidad, Marena; Nisonoff, Hunter; Chen, Kai; Lahiri, Arushi; Boger, Ron; Jacobsen, Steve; Banfield, Jillian F; Doudna, Jennifer A Nucleic acids research 37971304 2023 Dec 11
Combinatorial development of nebulized mRNA delivery formulations for the lungs. Jiang, Allen Y; Witten, Jacob; Raji, Idris O; Eweje, Feyisayo; MacIsaac, Corina; Meng, Sabrina; Oladimeji, Favour A; Hu, Yizong; Manan, Rajith S; Langer, Robert; Anderson, Daniel G Nature nanotechnology 37985700 2023 Nov 20
Self-delivering, chemically modified CRISPR RNAs for AAV co-delivery and genome editing in vivo. Zhang, Han; Kelly, Karen; Lee, Jonathan; Echeverria, Dimas; Cooper, David; Panwala, Rebecca; Amrani, Nadia; Chen, Zexiang; Gaston, Nicholas; Wagh, Atish; Newby, Gregory A; Xie, Jun; Liu, David R; Gao, Guangping; Wolfe, Scot A; Khvorova, Anastasia; Watts, Jonathan K; Sontheimer, Erik J Nucleic acids research 38033325 2023 Nov 30
Shuttle peptide delivers base editor RNPs to rhesus monkey airway epithelial cells in vivo. Kulhankova, Katarina; Traore, Soumba; Cheng, Xue; Benk-Fortin, Hadrien; Hallée, Stéphanie; Harvey, Mario; Roberge, Joannie; Couture, Frédéric; Kohli, Sajeev; Gross, Thomas J; Meyerholz, David K; Rettig, Garrett R; Thommandru, Bernice; Kurgan, Gavin; Wohlford-Lenane, Christine; Hartigan-O'Connor, Dennis J; Yates, Bradley P; Newby, Gregory A; Liu, David R; Tarantal, Alice F; Guay, David; McCray Jr, Paul B Nature communications 38052872 2023 Dec 05
Development of a human glioblastoma model using humanized DRAG mice for immunotherapy. Srivastava, Rashmi; Labani-Motlagh, Alireza; Chen, Apeng; Bohorquez, Jose Alejandro; Qin, Bin; Dodda, Meghana; Yang, Fan; Ansari, Danish; Patel, Sahil; Ji, Honglong; Trasti, Scott; Chao, Yapeng; Patel, Yash; Zou, Han; Hu, Baoli; Yi, Guohua Antibody therapeutics 38075240 2023 Oct
Harnessing non-Watson-Crick's base pairing to enhance CRISPR effectors cleavage activities and enable gene editing in mammalian cells. Gao, Shuliang; Guan, Huiwen; Bloomer, Hanan; Wich, Douglas; Song, Donghui; Khirallah, Jennifer; Ye, Zhongfeng; Zhao, Yu; Chen, Mengting; Xu, Chutian; Liu, Lihan; Xu, Qiaobing Proceedings of the National Academy of Sciences of the United States of America 38150477 2024 Jan 09
Tumor-tropic Trojan horses: Using mesenchymal stem cells as cellular nanotheranostics. Rosu, Ana; Ghaemi, Behnaz; Bulte, Jeff W M; Shakeri-Zadeh, Ali Theranostics 38169524 2024
An AAV capsid reprogrammed to bind human Transferrin Receptor mediates brain-wide gene delivery. Huang, Qin; Chan, Ken Y; Lou, Shan; Keyes, Casey; Wu, Jason; Botticello-Romero, Nuria R; Zheng, Qingxia; Johnston, Jencilin; Mills, Allan; Brauer, Pamela P; Clouse, Gabrielle; Pacouret, Simon; Harvey, John W; Beddow, Thomas; Hurley, Jenna K; Tobey, Isabelle G; Powell, Megan; Chen, Albert T; Barry, Andrew J; Eid, Fatma-Elzahraa; Chan, Yujia A; Deverman, Benjamin E bioRxiv : the preprint server for biology 38187643 2023 Dec 22
Engineered virus-like particles for transient delivery of prime editor ribonucleoprotein complexes in vivo. An, Meirui; Raguram, Aditya; Du, Samuel W; Banskota, Samagya; Davis, Jessie R; Newby, Gregory A; Chen, Paul Z; Palczewski, Krzysztof; Liu, David R Nature biotechnology 38191664 2024 Jan 08
Mechanosensitive genomic enhancers potentiate the cellular response to matrix stiffness. Cosgrove, Brian D; Bounds, Lexi R; Taylor, Carson Key; Su, Alan L; Rizzo, Anthony J; Barrera, Alejandro; Crawford, Gregory E; Hoffman, Brenton D; Gersbach, Charles A bioRxiv : the preprint server for biology 38260455 2024 Jan 10

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