Somatic Cell Genome Editing (93)
Publication TitleAuthorsJournalPublication DatePage NoPubMedID
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, JennieKidney360.2020 Mar;32656538
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, GuangpingNature communications.2020 Jun 30;32606306
Programmable m6A modification of cellular RNAs with a Cas13-directed methyltransferase.Wilson, Christopher; Chen, Peter J; Miao, Zhuang; Liu, David RNature biotechnology.2020 Jun 29;32601430
Polymeric vehicles for nucleic acid delivery.Piotrowski-Daspit, Alexandra S; Kauffman, Amy C; Bracaglia, Laura G; Saltzman, W MarkAdvanced drug delivery reviews.2020 Jun 23;32585159
Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors.Anzalone, Andrew V; Koblan, Luke W; Liu, David RNature biotechnology.2020 Jun 22;32572269
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, SamanthNature biotechnology.2020 Jun 15;32541958
The developing toolkit of continuous directed evolution.Morrison, Mary S; Podracky, Christopher J; Liu, David RNature chemical biology.2020 Jun;32444838
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 RNature biotechnology.2020 Mar 16;32433547
Advances in genome editing for genetic hearing loss.Ding, Ning; Lee, Sangsin; Lieber-Kotz, Matan; Yang, Jie; Gao, XueAdvanced drug delivery reviews.2020 May 07;32387678
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, ShaoqinJournal of controlled release : official journal of the Controlled Release Society.2020 May 05;32380204
Once upon a dish: engineering multicellular systems.Haase, Kristina; Freedman, Benjamin SDevelopment (Cambridge, England).2020 May 04;32366376
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 RMolecular therapy : the journal of the American Society of Gene Therapy.2020 Jun 03;32348718
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, WenNature communications.2020 Apr 24;32332735
Myogenic Progenitor Cell Lineage Specification by CRISPR/Cas9-Based Transcriptional Activators.Kwon, Jennifer B; Vankara, Ashish; Ettyreddy, Adarsh R; Bohning, Joel D; Gersbach, Charles AStem cell reports.2020 05 12;32330446
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, SuneetCell stem cell.2020 Jun 04;32320679
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, NenadToxicological sciences : an official journal of the Society of Toxicology.2020 Jul 01;32294208
Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases.Wu, Shao-Shuai; Li, Qing-Cui; Yin, Chang-Qing; Xue, Wen; Song, Chun-QingTheranostics.2020;32292501
CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors.Wang, Dan; Zhang, Feng; Gao, GuangpingCell.2020 Apr 02;32243786
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, JiangbingAdvanced science (Weinheim, Baden-Wurttemberg, Germany).2020 Mar;32154067
Can we mimic skeletal muscles for novel drug discovery?Broer, Torie; Khodabukus, Alastair; Bursac, NenadExpert opinion on drug discovery.2020 Mar 03;32122176
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 MMethods in molecular biology (Clifton, N.J.).2020;32088877
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, JunghaeACS synthetic biology.2020 Mar 20;32068391
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-CarnevaliNature.2020 02;32051592
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 MMolecules (Basel, Switzerland).2020 Feb 08;32046275
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 RNature biotechnology.2020 Apr;32042170
Evaluation and minimization of Cas9-independent off-target DNA editing by cytosine base editors.Doman, Jordan L; Raguram, Aditya; Newby, Gregory A; Liu, David RNature biotechnology.2020 Feb 10;32042165
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 MarkProceedings of the National Academy of Sciences of the United States of America.2020 Feb 18;32015123
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 MarkNano letters.2020 Feb 12;32003222
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 TravisCirculation research.2020 Jan 31;31999535
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 CaliniScience.2020 Jan 24;31958756
Ex vivo cell-based CRISPR/Cas9 genome editing for therapeutic applications.Li, Yamin; Glass, Zachary; Huang, Mingqian; Chen, Zheng-Yi; Xu, QiaobingBiomaterials.2020 Mar;31945616
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 RNature biomedical engineering.2020 Jan;31937940
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 KHuman gene therapy methods.2019 12;31855083
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, WenNature biomedical engineering.2020 Jan;31740768
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, WeizhiAdvanced science (Weinheim, Baden-Wurttemberg, Germany).2019 Nov 06;31728271
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 EPloS one.2019;31725765
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.2019 11 12;31698466
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, YanliMolecular cell.2019 12 19;31668930
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.2019 10 28;31659165
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 MarkJournal of controlled release : official journal of the Controlled Release Society.2019 Nov 28;31654688
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 RNature.2019 12;31634902
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 MAfrican journal of laboratory medicine.2019;31534913
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, ShaoqinNature nanotechnology.2019 Oct;31501532
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 JRNA (New York, N.Y.).2019 11;31439808
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, JakubThe Journal of investigative dermatology.2020 Feb;31437443
Preparation of a DNA Standard for Quantitative Polymerase Chain Reaction (qPCR).Lock, Martin; Korn, Michael; Wilson, James; Sena-Esteves, Miguel; Gao, GuangpingCold Spring Harbor protocols.2019 Aug 01;31371468
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, GuangpingCold Spring Harbor protocols.2019 Aug 01;31371467
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; JNature genetics.2019 08;31367015
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, QiaobingBiomacromolecules.2019 09 09;31342740
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 RNature biotechnology.2019 Jul 22;31332326
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-HyuckNature communications.2019 07 04;31273195
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 RNature communications.2019 07 02;31266953
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, MingAdvanced materials (Deerfield Beach, Fla.).2019 Jun 19;31215123
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, WenHepatology (Baltimore, Md.).2019 Jun 12;31188495
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, GuangpingJCI insight.2019 May 21;31112525
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 RNature biotechnology.2019 Jun;31110355
Development of hRad51-Cas9 nickase fusions that mediate HDR without double-stranded breaks.Rees, Holly A; Yeh, Wei-Hsi; Liu, David RNature communications.2019 05 17;31101808
Analysis and minimization of cellular RNA editing by DNA adenine base editors.Rees, Holly A; Wilson, Christopher; Doman, Jordan L; Liu, David RScience advances.2019 May;31086823
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 LHuman gene therapy.2019 Jun 17;31072208
Purification of the Recombinant Adenovirus by Cesium Chloride Gradient Centrifugation.Su, Qin; Sena-Esteves, Miguel; Gao, GuangpingCold Spring Harbor protocols.2019 05 01;31043560
Release of the Cloned Recombinant Adenovirus Genome for Rescue and Expansion.Su, Qin; Sena-Esteves, Miguel; Gao, GuangpingCold Spring Harbor protocols.2019 05 01;31043559
Construction of Recombinant Adenovirus Genomes by Direct Cloning.Zhou, Xiangyang; Sena-Esteves, Miguel; Gao, GuangpingCold Spring Harbor protocols.2019 05 01;31043558
Nucleic Acid Therapeutics for Neurological Diseases.Watts, Jonathan K; Brown, Robert H; Khvorova, AnastasiaNeurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics.2019 04;31037650
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 EScience (New York, N.Y.).2019 04 19;31000663
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, WenGenome medicine.2019 04 16;30987660
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 ZNature chemical biology.2019 05;30936501
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 ANucleic acids research.2019 Mar 20;30892626
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 CImmunity.2019 Mar 19;30850342
Intravenous Infusion of AAV for Widespread Gene Delivery to the Nervous System.Gessler, Dominic J; Tai, Phillip W L; Li, Jia; Gao, GuangpingMethods in molecular biology (Clifton, N.J.).2019;30783972
Quantitative and Digital Droplet-Based AAV Genome Titration.Sanmiguel, Julio; Gao, Guangping; Vandenberghe, Luk HMethods in molecular biology (Clifton, N.J.).2019;30783968
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, LotharScientific reports.2019 Feb 07;30733567
Adeno-associated virus vector as a platform for gene therapy delivery.Wang, Dan; Tai, Phillip W L; Gao, GuangpingNature reviews. Drug discovery.2019 05;30710128
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, MiMolecular therapy : the journal of the American Society of Gene Therapy.2019 03 06;30704961
Enhancing the In Vitro and In Vivo Stabilities of Polymeric Nucleic Acid Delivery Nanosystems.Wang, Yuyuan; Ye, Mingzhou; Xie, Ruosen; Gong, ShaoqinBioconjugate chemistry.2019 02 20;30592619
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, QiaobingAccounts of chemical research.2019 Mar 19;30586281
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 JMolecular cell.2018 Dec 18;30581144
Illuminating the genome-wide activity of genome editors for safe and effective therapeutics.Cheng, Yong; Tsai, Shengdar QGenome biology.2018 12 22;30577870
Intracellular Delivery of His-Tagged Genome-Editing Proteins Enabled by Nitrilotriacetic Acid-Containing Lipidoid Nanoparticles.Li, Yamin; Li, Alice Chukun; Xu, QiaobingAdvanced healthcare materials.2019 Mar;30565897
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, LotharNature communications.2018 Nov 15;30442934
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, WeiqingMolecular therapy. Methods & clinical development.2018 Dec 14;30406154
Understanding and repurposing CRISPR-mediated alternative splicing.Smith, Jordan L; Mou, Haiwei; Xue, WenGenome biology.2018 11 06;30400804
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, GuangpingMolecular therapy. Methods & clinical development.2018 Dec 14;30397628
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, FlorianHuman gene therapy.2019 May;30358470
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 QNature protocols.2018 11;30341435
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 JGenome biology.2018 Sep 19;30231914
The temporal landscape of recursive splicing during Pol II transcription elongation in human cells.Zhang, Xiao-Ou; Fu, Yu; Mou, Haiwei; Xue, Wen; Weng, ZhipingPLoS genetics.2018 08;30148885
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, GuangpingNature biotechnology.2018 Oct;30102296
Clinical Evaluations of Toxicity and Efficacy of Nanoparticle-Mediated Gene Therapy.Liang, Xiao; Liu, Li; Wei, Yu-Quan; Gao, Guang-Ping; Wei, Xia-WeiHuman gene therapy.2018 11;29893153
H3Africa: current perspectives.Mulder, Nicola; Abimiku, Alash'le; Adebamowo, Sally N; de Vries, Jantina; Matimba, Alice; Olowoyo, Paul; Ramsay, Michele; Skelton, Michelle; Stein, Dan JPharmacogenomics and personalized medicine.2018;29692621
Monitoring Lentivirus Vector Stocks for Replication-Competent Viruses.Sena-Esteves, Miguel; Gao, GuangpingCold Spring Harbor protocols.2018 04 02;29610364
Titration of Lentivirus Vectors.Sena-Esteves, Miguel; Gao, GuangpingCold Spring Harbor protocols.2018 04 02;29610363
Production of High-Titer Retrovirus and Lentivirus Vectors.Sena-Esteves, Miguel; Gao, GuangpingCold Spring Harbor protocols.2018 04 02;29610362
Research capacity. Enabling the genomic revolution in Africa.H3Africa ConsortiumScience (New York, N.Y.)2014 Jun 201346-824948725