Somatic Cell Genome Editing (54)
Publication TitleAuthorsJournalPublication DatePage NoPubMedID
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
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
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
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
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
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
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
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
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