Somatic Cell Genome Editing (SCGE)
Somatic Cell Genome Editing (SCGE)
Program Publications
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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; |
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| 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 |
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| 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 |
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| 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 |
| Genome-wide profiling of prime editor off-target sites in vitro and in vivo using PE-tag. | Liang, Shun-Qing; Liu, Pengpeng; Ponnienselvan, Karthikeyan; Suresh, Sneha; Chen, Zexiang; Kramme, Christian; Chatterjee, Pranam; Zhu, Lihua Julie; Sontheimer, Erik J; Xue, Wen; Wolfe, Scot A | Nature methods | 37156841 | 2023 Jun |
| 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 |
| CFTR-rich ionocytes mediate chloride absorption across airway epithelia. | Lei, Lei; Traore, Soumba; Romano Ibarra, Guillermo S; Karp, Philip H; Rehman, Tayyab; Meyerholz, David K; Zabner, Joseph; Stoltz, David A; Sinn, Patrick L; Welsh, Michael J; McCray Jr, Paul B; Thornell, Ian M | The Journal of clinical investigation | 37581935 | 2023 Oct 16 |
| 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 |
| A marmoset brain cell census reveals regional specialization of cellular identities. | Krienen, Fenna M; Levandowski, Kirsten M; Zaniewski, Heather; Del Rosario, Ricardo C H; Schroeder, Margaret E; Goldman, Melissa; Wienisch, Martin; Lutservitz, Alyssa; Beja-Glasser, Victoria F; Chen, Cindy; Zhang, Qiangge; Chan, Ken Y; Li, Katelyn X; Sharma, Jitendra; McCormack, Dana; Shin, Tay Won; Harrahill, Andrew; Nyase, Eric; Mudhar, Gagandeep; Mauermann, Abigail; Wysoker, Alec; Nemesh, James; Kashin, Seva; Vergara, Josselyn; Chelini, Gabriele; Dimidschstein, Jordane; Berretta, Sabina; Deverman, Benjamin E; Boyden, Ed; McCarroll, Steven A; Feng, Guoping | Science advances | 37824615 | 2023 Oct 13 |
| 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 |
| Non-canonical amino acid incorporation into AAV5 capsid enhances lung transduction in mice. | Chang, Hao; Du, Ailing; Jiang, Jun; Ren, Lingzhi; Liu, Nan; Zhou, Xuntao; Liang, Jialing; Gao, Guangping; Wang, Dan | Molecular therapy. Methods & clinical development | 37886602 | 2023 Dec 14 |
| 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 |
| Genomic characterization of SARS-CoV-2 from Uganda using MinION nanopore sequencing. | Kia, Praiscillia; Katagirya, Eric; Kakembo, Fredrick Elishama; Adera, Doreen Ato; Nsubuga, Moses Luutu; Yiga, Fahim; Aloyo, Sharley Melissa; Aujat, Brendah Ronah; Anguyo, Denis Foe; Katabazi, Fred Ashaba; Kigozi, Edgar; Joloba, Moses L; Kateete, David Patrick | Scientific reports | 37993530 | 2023 Nov 22 |
| 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 |
| Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophy. | Alves, Christiano R R; Ha, Leillani L; Yaworski, Rebecca; Sutton, Emma R; 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, Ras | Nature biomedical engineering | 38057426 | 2024 Feb |
| 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 |
| Protein-based nanoparticles for therapeutic nucleic acid delivery. | Eweje, Feyisayo; Walsh, Michelle L; Ahmad, Kiran; Ibrahim, Vanessa; Alrefai, Assma; Chen, Jiaxuan; Chaikof, Elliot L | Biomaterials | 38181574 | 2024 Mar |
| 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 |
| Validated assays for the quantification of C9orf72 human pathology. | Salomonsson, S E; Maltos, A M; Gill, K; Aladesuyi Arogundade, O; Brown, K A; Sachdev, A; Sckaff, M; Lam, K J K; Fisher, I J; Chouhan, R S; Van Laar, V S; Marley, C B; McLaughlin, I; Bankiewicz, K S; Tsai, Y-C; Conklin, B R; Clelland, C D | Scientific reports | 38191789 | 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 |
| AAV-based gene editing of type 1 collagen mutation to treat osteogenesis imperfecta. | Yang, Yeon-Suk; Sato, Tadatoshi; Chaugule, Sachin; Ma, Hong; Xie, Jun; Gao, Guangping; Shim, Jae-Hyuck | Molecular therapy. Nucleic acids | 38261950 | 2024 Mar 12 |
| Advancements in therapeutic development: kidney organoids and organs on a chip. | Tabibzadeh, Nahid; Morizane, Ryuji | Kidney international | 38296026 | 2024 Apr |
| Phage-assisted evolution of highly active cytosine base editors with enhanced selectivity and minimal sequence context preference. | Zhang, Emily; Neugebauer, Monica E; Krasnow, Nicholas A; Liu, David R | Nature communications | 38402281 | 2024 Feb 24 |
| Cationic cholesterol-dependent LNP delivery to lung stem cells, the liver, and heart. | Radmand, Afsane; Kim, Hyejin; Beyersdorf, Jared; Dobrowolski, Curtis N; Zenhausern, Ryan; Paunovska, Kalina; Huayamares, Sebastian G; Hua, Xuanwen; Han, Keyi; Loughrey, David; Hatit, Marine Z C; Del Cid, Ada; Ni, Huanzhen; Shajii, Aram; Li, Andrea; Muralidharan, Abinaya; Peck, Hannah E; Tiegreen, Karen E; Jia, Shu; Santangelo, Philip J; Dahlman, James E | Proceedings of the National Academy of Sciences of the United States of America | 38437539 | 2024 Mar 12 |
| Targeted nonviral delivery of genome editors in vivo. | Tsuchida, Connor A; Wasko, Kevin M; Hamilton, Jennifer R; Doudna, Jennifer A | Proceedings of the National Academy of Sciences of the United States of America | 38437567 | 2024 Mar 12 |
| A humanized mouse model for adeno-associated viral gene therapy. | Barzi, Mercedes; Chen, Tong; Gonzalez, Trevor J; Pankowicz, Francis P; Oh, Seh Hoon; Streff, Helen L; Rosales, Alan; Ma, Yunhan; Collias, Sabrina; Woodfield, Sarah E; Diehl, Anna Mae; Vasudevan, Sanjeev A; Galvan, Thao N; Goss, John; Gersbach, Charles A; Bissig-Choisat, Beatrice; Asokan, Aravind; Bissig, Karl-Dimiter | Nature communications | 38438373 | 2024 Mar 04 |
| Bacteriophage therapy for the treatment of Mycobacterium tuberculosis infections in humanized mice. | Yang, Fan; Labani-Motlagh, Alireza; Bohorquez, Jose Alejandro; Moreira, Josimar Dornelas; Ansari, Danish; Patel, Sahil; Spagnolo, Fabrizio; Florence, Jon; Vankayalapati, Abhinav; Sakai, Tsuyoshi; Sato, Osamu; Ikebe, Mitsuo; Vankayalapati, Ramakrishna; Dennehy, John J; Samten, Buka; Yi, Guohua | Communications biology | 38461214 | 2024 Mar 09 |
| Hachiman is a genome integrity sensor. | Tuck, Owen T; Adler, Benjamin A; Armbruster, Emily G; Lahiri, Arushi; Hu, Jason J; Zhou, Julia; Pogliano, Joe; Doudna, Jennifer A | bioRxiv : the preprint server for biology | 38464307 | 2024 Feb 29 |
| Long-term efficacy and safety of cardiac genome editing for catecholaminergic polymorphic ventricular tachycardia. | Moore, Oliver M; Aguilar-Sanchez, Yuriana; Lahiri, Satadru K; Hulsurkar, Mohit M; Alberto Navarro-Garcia, J; Word, Tarah A; Keefe, Joshua A; Barazi, Dean; Munivez, Elda M; Moore, Charles T; Parthasarathy, Vaidya; Davidson, Jaysón; Lagor, William R; Park, So Hyun; Bao, Gang; Miyake, Christina Y; Wehrens, Xander H T | The journal of cardiovascular aging | 38464671 | 2024 Jan |
| A Novel Humanized Mouse Model for HIV and Tuberculosis Co-infection Studies. | Bohórquez, José Alejandro; Adduri, Sitaramaraju; Ansari, Danish; John, Sahana; Florence, Jon; Adejare, Omoyeni; Singh, Gaurav; Konduru, Nagarjun; Jagannath, Chinnaswamy; Yi, Guohua | bioRxiv : the preprint server for biology | 38496484 | 2024 Mar 07 |
| Precision Enhancement of CAR-NK Cells through Non-Viral Engineering and Highly Multiplexed Base Editing. | Wang, Minjing; Krueger, Joshua B; Gilkey, Alexandria K; Stelljes, Erin M; Kluesner, Mitchell G; Pomeroy, Emily J; Skeate, Joseph G; Slipek, Nicholas J; Lahr, Walker S; Vázquez, Patricia N Claudio; Zhao, Yueting; Eaton, Ella J; Laoharawee, Kanut; Webber, Beau R; Moriarity, Branden S | bioRxiv : the preprint server for biology | 38496503 | 2024 Mar 08 |
| Activation of the imprinted Prader-Willi Syndrome locus by CRISPR-based epigenome editing. | Rohm, Dahlia; Black, Joshua B; McCutcheon, Sean R; Barrera, Alejandro; Morone, Daniel J; Nuttle, Xander; de Esch, Celine E; Tai, Derek J C; Talkowski, Michael E; Iglesias, Nahid; Gersbach, Charles A | bioRxiv : the preprint server for biology | 38496583 | 2024 Mar 04 |
| Adeno-associated virus as a delivery vector for gene therapy of human diseases. | Wang, Jiang-Hui; Gessler, Dominic J; Zhan, Wei; Gallagher, Thomas L; Gao, Guangping | Signal transduction and targeted therapy | 38565561 | 2024 Apr 03 |
