Somatic Cell Genome Editing (208)
Publication TitleAuthorsJournalPublication DatePage NoPubMedID
Engineered pegRNAs improve prime editing efficiency.Nelson, James W; Randolph, Peyton B; Shen, Simon P; Everette, Kelcee A; Chen, Peter J; Anzalone, Andrew V; An, Meirui; Newby, Gregory A; Chen, Jonathan C; Hsu, Alvin; Liu, David RNature biotechnology2021 Oct 0434608327
In Vivo MRI Tracking of Tumor Vaccination and Antigen Presentation by Dendritic Cells.Bulte, Jeff W M; Shakeri-Zadeh, AliMolecular imaging and biology2021 Sep 2834581954
Light: A Magical Tool for Controlled Drug Delivery.Tao, Yu; Chan, Hon Fai; Shi, Bingyang; Li, Mingqiang; Leong, Kam WAdvanced functional materials2020 Dec 0134483808
Cortical overgrowth in a preclinical forebrain organoid model of CNTNAP2-associated autism spectrum disorder.de Jong, Job O; Llapashtica, Ceyda; Genestine, Matthieu; Strauss, Kevin; Provenzano, Frank; Sun, Yan; Zhu, Huixiang; Cortese, Giuseppe P; Brundu, Francesco; Brigatti, Karlla W; Corneo, Barbara; Migliori, Bianca; Tomer, Raju; Kushner, Steven A; Kellendonk, Christoph; Javitch, Jonathan A; Xu, Bin; Markx, SanderNature communications2021 09 0134471112
Flash Technology-Based Self-Assembly in Nanoformulation: From Fabrication to Biomedical Applications.Hu, Hanze; Yang, Chao; Li, Mingqiang; Shao, Dan; Mao, Hai-Quan; Leong, Kam WMaterials today (Kidlington, England)2021 Jan-Feb34421329
Synthetic Biology Approaches for Engineering Next-Generation Adenoviral Gene Therapies.Collins, Logan Thrasher; Curiel, David TACS nano2021 09 2834415739
Easy-Prime: a machine learning-based prime editor design tool.Li, Yichao; Chen, Jingjing; Tsai, Shengdar Q; Cheng, YongGenome biology2021 08 1934412673
Bioengineered optogenetic model of human neuromuscular junction.Vila, Olaia F; Chavez, Miguel; Ma, Stephen P; Yeager, Keith; Zholudeva, Lyandysha V; Colón-Mercado, Jennifer M; Qu, Yihuai; Nash, Trevor R; Lai, Carmen; Feliciano, Carissa M; Carter, Matthew; Kamm, Roger D; Judge, Luke M; Conklin, Bruce R; Ward, Michael E; McDevitt, Todd C; Vunjak-Novakovic, GordanaBiomaterials2021 0934403849
A Versatile and Robust Platform for the Scalable Manufacture of Biomimetic Nanovaccines.Hu, Hanze; Yang, Chao; Zhang, Fan; Li, Mingqiang; Tu, Zhaoxu; Mu, Lizhong; Dawulieti, Jianati; Lao, Yeh-Hsing; Xiao, Zixuan; Yan, Huize; Sun, Wen; Shao, Dan; Leong, Kam WAdvanced science (Weinheim, Baden-Wurttemberg, Germany)2021 0834386315
DNA interference states of the hypercompact CRISPR-CasΦ effector.Pausch, Patrick; Soczek, Katarzyna M; Herbst, Dominik A; Tsuchida, Connor A; Al-Shayeb, Basem; Banfield, Jillian F; Nogales, Eva; Doudna, Jennifer ANature structural & molecular biology2021 0834381246
Actinin BioID reveals sarcomere crosstalk with oxidative metabolism through interactions with IGF2BP2.Ladha, Feria A; Thakar, Ketan; Pettinato, Anthony M; Legere, Nicholas; Ghahremani, Shahnaz; Cohn, Rachel; Romano, Robert; Meredith, Emily; Chen, Yu-Sheng; Hinson, J TravisCell reports2021 Aug 1034380038
Transgenic mice for in vivo epigenome editing with CRISPR-based systems.Gemberling, Matthew P; Siklenka, Keith; Rodriguez, Erica; Tonn-Eisinger, Katherine R; Barrera, Alejandro; Liu, Fang; Kantor, Ariel; Li, Liqing; Cigliola, Valentina; Hazlett, Mariah F; Williams, Courtney A; Bartelt, Luke C; Madigan, Victoria J; Bodle, Josephine C; Daniels, Heather; Rouse, Douglas C; Hilton, Isaac B; Asokan, Aravind; Ciofani, Maria; Poss, Kenneth D; Reddy, Timothy E; West, Anne E; Gersbach, Charles ANature methods2021 0834341582
Label-free imaging for quality control of cardiomyocyte differentiation.Qian, Tongcheng; Heaster, Tiffany M; Houghtaling, Angela R; Sun, Kexin; Samimi, Kayvan; Skala, Melissa CNature communications2021 07 2834321477
Vector Strategies to Actualize B Cell-Based Gene Therapies.Jeske, Amanda M; Boucher, Paul; Curiel, David T; Voss, James EJournal of immunology (Baltimore, Md. : 1950)2021 08 0134321286
External stimuli-responsive nanoparticles for spatially and temporally controlled delivery of CRISPR-Cas genome editors.Xie, Ruosen; Wang, Yuyuan; Gong, ShaoqinBiomaterials science2021 Sep 1434286726
Recent innovations in fluorescence lifetime imaging microscopy for biology and medicine.Datta, Rupsa; Gillette, Amani; Stefely, Matthew; Skala, Melissa CJournal of biomedical optics2021 0734247457
Receptor Switching in Newly Evolved Adeno-associated Viruses.Havlik, L Patrick; Das, Anshuman; Mietzsch, Mario; Oh, Daniel K; Ark, Jonathan; McKenna, Robert; Agbandje-McKenna, Mavis; Asokan, AravindJournal of virology2021 09 0934232726
Mucopolysaccharidoses type I gene therapy.Hurt, Sarah C; Dickson, Patricia I; Curiel, David TJournal of inherited metabolic disease2021 Jun 2934189746
Efficient C•G-to-G•C base editors developed using CRISPRi screens, target-library analysis, and machine learning.Koblan, Luke W; Arbab, Mandana; Shen, Max W; Hussmann, Jeffrey A; Anzalone, Andrew V; Doman, Jordan L; Newby, Gregory A; Yang, Dian; Mok, Beverly; Replogle, Joseph M; Xu, Albert; Sisley, Tyler A; Weissman, Jonathan S; Adamson, Britt; Liu, David RNature biotechnology2021 Jun 2834183861
Gene editing in a Myo6 semi-dominant mouse model rescues auditory function.Xue, Yuanyuan; Hu, Xinde; Wang, Daqi; Li, Di; Li, Yige; Wang, Fang; Huang, Mingqian; Gu, Xi; Xu, Zhijiao; Zhou, Jinan; Wang, Jinghan; Chai, Renjie; Shen, Jun; Chen, Zheng-Yi; Li, Geng-Lin; Yang, Hui; Li, Huawei; Zuo, Erwei; Shu, YilaiMolecular therapy : the journal of the American Society of Gene Therapy2021 Jun 2434174443
Controlling CRISPR with small molecule regulation for somatic cell genome editing.Khajanchi, Namita; Saha, KrishanuMolecular therapy : the journal of the American Society of Gene Therapy2021 Jun 2434174442
In vivo targeted delivery of nucleic acids and CRISPR genome editors enabled by GSH-responsive silica nanoparticles.Wang, Yuyuan; Shahi, Pawan K; Wang, Xiuxiu; Xie, Ruosen; Zhao, Yi; Wu, Min; Roge, Seth; Pattnaik, Bikash R; Gong, ShaoqinJournal of controlled release : official journal of the Controlled Release Society2021 Jun 2334174352
Base editing of haematopoietic stem cells rescues sickle cell disease in mice.Newby, Gregory A; Yen, Jonathan S; Woodard, Kaitly J; Mayuranathan, Thiyagaraj; Lazzarotto, Cicera R; Li, Yichao; Sheppard-Tillman, Heather; Porter, Shaina N; Yao, Yu; Mayberry, Kalin; Everette, Kelcee A; Jang, Yoonjeong; Podracky, Christopher J; Thaman, Elizabeth; Lechauve, Christophe; Sharma, Akshay; Henderson, Jordana M; Richter, Michelle F; Zhao, Kevin T; Miller, Shannon M; Wang, Tina; Koblan, Luke W; McCaffrey, Anton P; Tisdale, John F; Kalfa, Theodosia A; Pruett-Miller, Shondra M; Tsai, Shengdar Q; Weiss, Mitchell J; Liu, David RNature2021 Jul34079130
Escaping the endosome: assessing cellular trafficking mechanisms of non-viral vehicles.Xu, Emily; Saltzman, W Mark; Piotrowski-Daspit, Alexandra SJournal of controlled release : official journal of the Controlled Release Society2021 07 1034077782
Targeted delivery of CRISPR-Cas9 and transgenes enables complex immune cell engineering.Hamilton, Jennifer R; Tsuchida, Connor A; Nguyen, David N; Shy, Brian R; McGarrigle, E Riley; Sandoval Espinoza, Cindy R; Carr, Daniel; Blaeschke, Franziska; Marson, Alexander; Doudna, Jennifer ACell reports2021 Jun 0134077734
SARS-CoV-2 infection induces the activation of tissue factor-mediated coagulation via activation of acid sphingomyelinase.Wang, Jue; Pendurthi, Usha R; Yi, Guohua; Rao, L Vijaya MohanBlood2021 07 2934075401
Generation and Genetic Correction of USH2A c.2299delG Mutation in Patient-Derived Induced Pluripotent Stem Cells.Liu, Xuezhong; Lillywhite, Justin; Zhu, Wenliang; Huang, Zaohua; Clark, Anna M; Gosstola, Nicholas; Maguire, Colin T; Dykxhoorn, Derek; Chen, Zheng-Yi; Yang, JunGenes2021 05 2534070435
Electrophoretic mobility shift as a molecular beacon-based readout for miRNA detection.Oliveira-Jr, Getulio P; Barbosa, Raquel H; Thompson, Lauren; Pinckney, Brandy; Murphy-Thornley, Moriah; Lu, Shulin; Jones, Jennifer; Hansen, Clinton H; Tigges, John; Wong, Wesley P; Ghiran, Ionita CBiosensors & bioelectronics2021 Oct 0134062334
Quantification of Cellular Densities and Antigenic Properties using Magnetic Levitation.Thompson, Lauren; Pinckney, Brandy; Lu, Shulin; Gregory, Mark; Tigges, John; Ghiran, IonitaJournal of visualized experiments : JoVE2021 05 1734057455
Vectored Immunotherapeutics for Infectious Diseases: Can rAAVs Be The Game Changers for Fighting Transmissible Pathogens?Zhan, Wei; Muhuri, Manish; Tai, Phillip W L; Gao, GuangpingFrontiers in immunology202134046041
Efficient embryonic homozygous gene conversion via RAD51-enhanced interhomolog repair.Wilde, Jonathan J; Aida, Tomomi; Del Rosario, Ricardo C H; Kaiser, Tobias; Qi, Peimin; Wienisch, Martin; Zhang, Qiangge; Colvin, Steven; Feng, GuopingCell2021 Jun 1034043941
Novel Combinatorial MicroRNA-Binding Sites in AAV Vectors Synergistically Diminish Antigen Presentation and Transgene Immunity for Efficient and Stable Transduction.Muhuri, Manish; Zhan, Wei; Maeda, Yukiko; Li, Jia; Lotun, Anoushka; Chen, Jennifer; Sylvia, Katelyn; Dasgupta, Ishani; Arjomandnejad, Motahareh; Nixon, Thomas; Keeler, Allison M; Manokaran, Sangeetha; He, Ran; Su, Qin; Tai, Phillip W L; Gao, GuangpingFrontiers in immunology202133995418
Sarcomere function activates a p53-dependent DNA damage response that promotes polyploidization and limits in vivo cell engraftment.Pettinato, Anthony M; Yoo, Dasom; VanOudenhove, Jennifer; Chen, Yu-Sheng; Cohn, Rachel; Ladha, Feria A; Yang, Xiulan; Thakar, Ketan; Romano, Robert; Legere, Nicolas; Meredith, Emily; Robson, Paul; Regnier, Michael; Cotney, Justin L; Murry, Charles E; HinsCell reports2021 May 0433951429
Integrating Biomaterials and Genome Editing Approaches to Advance Biomedical Science.Abdeen, Amr A; Cosgrove, Brian D; Gersbach, Charles A; Saha, KrishanuAnnual review of biomedical engineering2021 07 1333909475
VectorMOD: Method for Bottom-Up Proteomic Characterization of rAAV Capsid Post-Translational Modifications and Vector Impurities.Rumachik, Neil G; Malaker, Stacy A; Paulk, Nicole KFrontiers in immunology202133868302
Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice.Liu, Pengpeng; Liang, Shun-Qing; Zheng, Chunwei; Mintzer, Esther; Zhao, Yan G; Ponnienselvan, Karthikeyan; Mir, Aamir; Sontheimer, Erik J; Gao, Guangping; Flotte, Terence R; Wolfe, Scot A; Xue, WenNature communications2021 04 0933837189
The NIH Somatic Cell Genome Editing program.Saha, Krishanu; Sontheimer, Erik J; Brooks, P J; Dwinell, Melinda R; Gersbach, Charles A; Liu, David R; Murray, Stephen A; Tsai, Shengdar Q; Wilson, Ross C; Anderson, Daniel G; Asokan, Aravind; Banfield, Jillian F; Bankiewicz, Krystof S; Bao, Gang; Bulte, Jeff W M; Bursac, Nenad; Campbell, Jarryd M; Carlson, Daniel F; Chaikof, Elliot L; Chen, Zheng-Yi; Cheng, R Holland; Clark, Karl J; Curiel, David T; Dahlman, James E; Deverman, Benjamin E; Dickinson, Mary E; Doudna, Jennifer A; Ekker, Stephen C; Emborg, Marina E; Feng, Guoping; Freedman, Benjamin S; Gamm, David M; Gao, Guangping; Ghiran, Ionita C; Glazer, Peter M; Gong, Shaoqin; Heaney, Jason D; Hennebold, Jon D; Hinson, John T; Khvorova, Anastasia; Kiani, Samira; Lagor, William R; Lam, Kit S; Leong, Kam W; Levine, Jon E; Lewis, Jennifer A; Lutz, Cathleen M; Ly, Danith H; Maragh, Samantha; McCray Jr, Paul B; McDevitt, Todd C; Mirochnitchenko, Oleg; Morizane, Ryuji; Murthy, Niren; Prather, Randall S; Ronald, John A; Roy, Subhojit; Roy, Sushmita; Sabbisetti, Venkata; Saltzman, W Mark; Santangelo, Philip J; Segal, David J; Shimoyama, Mary; Skala, Melissa C; Tarantal, Alice F; Tilton, John C; Truskey, George A; Vandsburger, Moriel; Watts, Jonathan K; Wells, Kevin D; Wolfe, Scot A; Xu, Qiaobing; Xue, Wen; Yi, Guohua; Zhou, Jiangbing; SCGE ConsortiumNature2021 Apr33828315
Examining the evidence for extracellular RNA function in mammals.Gruner, Hannah N; McManus, Michael TNature reviews. Genetics2021 0733824487
PEGylation of poly(amine-co-ester) polyplexes for tunable gene delivery.Grun, Molly K; Suberi, Alexandra; Shin, Kwangsoo; Lee, Teresa; Gomerdinger, Victoria; Moscato, Zoe M; Piotrowski-Daspit, Alexandra S; Saltzman, W MarkBiomaterials2021 May33813260
Large-scale molecular epidemiological analysis of AAV in a cancer patient population.Qin, Wanru; Xu, Guangchao; Tai, Phillip W L; Wang, Chunmei; Luo, Li; Li, Chengjian; Hu, Xun; Xue, Jianxin; Lu, You; Zhou, Qiao; Wei, Qiang; Wen, Tianfu; Hu, Jiankun; Xiao, Yuanyuan; Yang, Li; Li, Weimin; Flotte, Terence R; Wei, Yuquan; Gao, GuangpingOncogene2021 Apr33782545
Increased CFTR expression and function from an optimized lentiviral vector for cystic fibrosis gene therapy.Marquez Loza, Laura I; Cooney, Ashley L; Dong, Qian; Randak, Christoph O; Rivella, Stefano; Sinn, Patrick L; McCray Jr, Paul BMolecular therapy. Methods & clinical development2021 Jun 1133768133
Nanoparticles containing constrained phospholipids deliver mRNA to liver immune cells in vivo without targeting ligands.Gan, Zubao; Lokugamage, Melissa P; Hatit, Marine Z C; Loughrey, David; Paunovska, Kalina; Sato, Manaka; Cristian, Ana; Dahlman, James EBioengineering & translational medicine2020 Sep33758781
SARS-CoV-2 infection of human iPSC-derived cardiac cells reflects cytopathic features in hearts of patients with COVID-19.Perez-Bermejo, Juan A; Kang, Serah; Rockwood, Sarah J; Simoneau, Camille R; Joy, David A; Silva, Ana C; Ramadoss, Gokul N; Flanigan, Will R; Fozouni, Parinaz; Li, Huihui; Chen, Pei-Yi; Nakamura, Ken; Whitman, Jeffrey D; Hanson, Paul J; McManus, Bruce M; Ott, Melanie; Conklin, Bruce R; McDevitt, Todd CScience translational medicine2021 04 2133723017
Prime editing in mice reveals the essentiality of a single base in driving tissue-specific gene expression.Gao, Pan; Lyu, Qing; Ghanam, Amr R; Lazzarotto, Cicera R; Newby, Gregory A; Zhang, Wei; Choi, Mihyun; Slivano, Orazio J; Holden, Kevin; Walker 2nd, John A; Kadina, Anastasia P; Munroe, Rob J; Abratte, Christian M; Schimenti, John C; Liu, David R; Tsai, Shengdar Q; Long, Xiaochun; Miano, Joseph MGenome biology2021 03 1633722289
Multicellular Systems to Translate Somatic Cell Genome Editors to Humans.Hernandez-Gordillo, Victor; Casolaro, Thomas Caleb; Ebrahimkhani, Mo R; Kiani, SamiraCurrent opinion in biomedical engineering2020 Dec33718690
Tissue-Engineered Skeletal Muscle Models to Study Muscle Function, Plasticity, and Disease.Khodabukus, AlastairFrontiers in physiology202133716768
Loss of sarcomeric proteins via upregulation of JAK/STAT signaling underlies interferon-γ-induced contractile deficit in engineered human myocardium.Zhan, Ren-Zhi; Rao, Lingjun; Chen, Zhaowei; Strash, Nicholas; Bursac, NenadActa biomaterialia2021 0533705988
A CRISPR-Cas9-integrase complex generates precise DNA fragments for genome integration.Jakhanwal, Shrutee; Cress, Brady F; Maguin, Pascal; Lobba, Marco J; Marraffini, Luciano A; Doudna, Jennifer ANucleic acids research2021 04 0633693715
Therapeutic RNA Delivery for COVID and Other Diseases.Dobrowolski, Curtis; Paunovska, Kalina; Hatit, Marine Z C; Lokugamage, Melissa P; Dahlman, James EAdvanced healthcare materials2021 Mar 0433661555
Lipid nanoparticle-mediated codelivery of Cas9 mRNA and single-guide RNA achieves liver-specific in vivo genome editing of Angptl3.Qiu, Min; Glass, Zachary; Chen, Jinjin; Haas, Mary; Jin, Xin; Zhao, Xuewei; Rui, Xuehui; Ye, Zhongfeng; Li, Yamin; Zhang, Feng; Xu, QiaobingProceedings of the National Academy of Sciences of the United States of America2021 Mar 0933649229
COVID-19 CG enables SARS-CoV-2 mutation and lineage tracking by locations and dates of interest.Chen, Albert Tian; Altschuler, Kevin; Zhan, Shing Hei; Chan, Yujia Alina; Deverman, Benjamin EeLife2021 02 2333620031
Engineering highly efficient backsplicing and translation of synthetic circRNAs.Meganck, Rita M; Liu, Jiacheng; Hale, Andrew E; Simon, Katherine E; Fanous, Marco M; Vincent, Heather A; Wilusz, Jeremy E; Moorman, Nathaniel J; Marzluff, William F; Asokan, AravindMolecular therapy. Nucleic acids2021 Mar 0533614232
Massively parallel assessment of human variants with base editor screens.Hanna, Ruth E; Hegde, Mudra; Fagre, Christian R; DeWeirdt, Peter C; Sangree, Annabel K; Szegletes, Zsofia; Griffith, Audrey; Feeley, Marissa N; Sanson, Kendall R; Baidi, Yossef; Koblan, Luke W; Liu, David R; Neal, James T; Doench, John GCell2021 02 1833606977
PrimeDesign software for rapid and simplified design of prime editing guide RNAs.Hsu, Jonathan Y; Grünewald, Julian; Szalay, Regan; Shih, Justine; Anzalone, Andrew V; Lam, Kin Chung; Shen, Max W; Petri, Karl; Liu, David R; Keith Joung, J; Pinello, LucaNature communications2021 02 1533589617
Soluble CD40L activates soluble and cell-surface integrins αvβ3, α5β1 and α4β1 by binding to the allosteric ligand-binding site (site 2).Takada, Yoko K; Shimoda, Michiko; Maverakis, Emanual; Felding, Brunie H; Cheng, R Holland; Takada, YoshikazuThe Journal of biological chemistry2021 Feb 0833571526
Viral vector platforms within the gene therapy landscape.Bulcha, Jote T; Wang, Yi; Ma, Hong; Tai, Phillip W L; Gao, GuangpingSignal transduction and targeted therapy2021 Feb 0833558455
Gene-based therapies for neurodegenerative diseases.Sun, Jichao; Roy, SubhojitNature neuroscience2021 0333526943
Exercise mimetics and JAK inhibition attenuate IFN-γ-induced wasting in engineered human skeletal muscle.Chen, Zhaowei; Li, Binjie; Zhan, Ren-Zhi; Rao, Lingjun; Bursac, NenadScience advances2021 Jan33523949
Nanoparticles for delivery of agents to fetal lungs.Ullrich, Sarah J; Freedman-Weiss, Mollie; Ahle, Samantha; Mandl, Hanna K; Piotrowski-Daspit, Alexandra S; Roberts, Katherine; Yung, Nicholas; Maassel, Nathan; Bauer-Pisani, Tory; Ricciardi, Adele S; Egan, Marie E; Glazer, Peter M; Saltzman, W Mark; Stitelman, David HActa biomaterialia2021 Jan 2133484911
Determining of canine position by multiple facial landmarks to achieve natural esthetics in complete denture treatment.Srimaneekarn, Natchalee; Arayapisit, Tawepong; Pooktuantong, Ornnicha; Cheng, Holland R; Soonsawad, PanThe Journal of prosthetic dentistry2021 Jan 1633468316
Precision genome editing using cytosine and adenine base editors in mammalian cells.Huang, Tony P; Newby, Gregory A; Liu, David RNature protocols2021 Feb33462442
CRISPR/Cas9 gene editing for curing sickle cell disease.Park, So Hyun; Bao, GangTransfusion and apheresis science : official journal of the World Apheresis Association : official journal of the European Society for Haemapheresis2021 Jan 1033455878
In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice.Koblan, Luke W; Erdos, Michael R; Wilson, Christopher; Cabral, Wayne A; Levy, Jonathan M; Xiong, Zheng-Mei; Tavarez, Urraca L; Davison, Lindsay M; Gete, Yantenew G; Mao, Xiaojing; Newby, Gregory A; Doherty, Sean P; Narisu, Narisu; Sheng, Quanhu; Krilow, Chad; Lin, Charles Y; Gordon, Leslie B; Cao, Kan; Collins, Francis S; Brown, Jonathan D; Liu, David RNature2021 Jan33408413
Overcoming innate immune barriers that impede AAV gene therapy vectors.Muhuri, Manish; Maeda, Yukiko; Ma, Hong; Ram, Sanjay; Fitzgerald, Katherine A; Tai, Phillip Wl; Gao, GuangpingThe Journal of clinical investigation2021 Jan 0433393506
Targeting de novo purine synthesis pathway via ADSL depletion impairs liver cancer growth by perturbing mitochondrial function.Jiang, Tingting; Sánchez-Rivera, Francisco J; Soto-Feliciano, Yadira M; Yang, Qiyuan; Song, Chun-Qing; Bhuatkar, Arjun; Haynes, Cole M; Hemann, Michael T; Xue, WenHepatology (Baltimore, Md.)2020 Dec 1733336367
Gene Regulatory Network Analysis and Engineering Directs Development and Vascularization of Multilineage Human Liver Organoids.Velazquez, Jeremy J; LeGraw, Ryan; Moghadam, Farzaneh; Tan, Yuqi; Kilbourne, Jacquelyn; Maggiore, Joseph C; Hislop, Joshua; Liu, Silvia; Cats, Davy; Chuva de Sousa Lopes, Susana M; Plaisier, Christopher; Cahan, Patrick; Kiani, Samira; Ebrahimkhani, Mo RCell systems2021 Jan 2033290741
Tools for experimental and computational analyses of off-target editing by programmable nucleases.Bao, X Robert; Pan, Yidan; Lee, Ciaran M; Davis, Timothy H; Bao, GangNature protocols.2020 Dec 07;33288953
Master Regulators and Cofactors of Human Neuronal Cell Fate Specification Identified by CRISPR Gene Activation Screens.Black, Joshua B; McCutcheon, Sean R; Dube, Shataakshi; Barrera, Alejandro; Klann, Tyler S; Rice, Grayson A; Adkar, Shaunak S; Soderling, Scott H; Reddy, Timothy E; Gersbach, Charles ACell reports.2020 Dec 01;33264623
Phage-assisted continuous and non-continuous evolution.Miller, Shannon M; Wang, Tina; Liu, David RNature protocols.2020 Dec;33199872
The once and future gene therapy.Bulaklak, Karen; Gersbach, Charles ANature communications.2020 11 16;33199717
Delayed urea differential enhancement CEST (dudeCEST)-MRI with T1 correction for monitoring renal urea handling.Shin, Soo Hyun; Wendland, Michael F; Vandsburger, Moriel HMagnetic resonance in medicine.2020 Nov 12;33180343
Poison Exon Splicing Regulates a Coordinated Network of SR Protein Expression during Differentiation and Tumorigenesis.Leclair, Nathan K; Brugiolo, Mattia; Urbanski, Laura; Lawson, Shane C; Thakar, Ketan; Yurieva, Marina; George, Joshy; Hinson, John Travis; Cheng, Albert; Graveley, Brenton R; Anczuków, OlgaMolecular cell.2020 11 19;33176162
In Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular Dystrophy.Kwon, Jennifer B; Ettyreddy, Adarsh R; Vankara, Ashish; Bohning, Joel D; Devlin, Garth; Hauschka, Stephen D; Asokan, Aravind; Gersbach, Charles AMolecular therapy. Methods & clinical development.2020 Dec 11;33145368
A Versatile Nonviral Delivery System for Multiplex Gene-Editing in the Liver.Gong, Jing; Wang, Hong-Xia; Lao, Yeh-Hsing; Hu, Hanze; Vatan, Naazanene; Guo, Jonathan; Ho, Tzu-Chieh; Huang, Dantong; Li, Mingqiang; Shao, Dan; Leong, Kam WAdvanced materials (Deerfield Beach, Fla.)2020 Nov33053221
3D kidney organoids for bench-to-bedside translation.Gupta, Navin; Dilmen, Emre; Morizane, RyujiJournal of molecular medicine (Berlin, Germany).2020 Oct 09;33034708
Development of a Cardiac Sarcomere Functional Genomics Platform to Enable Scalable Interrogation of Human TNNT2 Variants.Pettinato, Anthony M; Ladha, Feria A; Mellert, David J; Legere, Nicholas; Cohn, Rachel; Romano, Robert; Thakar, Ketan; Chen, Yu-Sheng; Hinson, J TravisCirculation.2020 Oct 07;33025817
COVID-19 CG: Tracking SARS-CoV-2 mutations by locations and dates of interest.Chen, Albert Tian; Altschuler, Kevin; Zhan, Shing Hei; Chan, Yujia Alina; Deverman, Benjamin EbioRxiv : the preprint server for biology.2020 Sep 28;32995794
Methods Matter: Standard Production Platforms for Recombinant AAV Produce Chemically and Functionally Distinct Vectors.Rumachik, Neil G; Malaker, Stacy A; Poweleit, Nicole; Maynard, Lucy H; Adams, Christopher M; Leib, Ryan D; Cirolia, Giana; Thomas, Dennis; Stamnes, Susan; Holt, Kathleen; Sinn, Patrick; May, Andrew P; Paulk, Nicole KMolecular therapy. Methods & clinical development.2020 Sep 11;32995354
Direct Functional Protein Delivery with a Peptide into Neonatal and Adult Mammalian Inner Ear In Vivo.Zhang, Kun; Cheng, Xiaoting; Zhao, Liping; Huang, Mingqian; Tao, Yong; Zhang, Hongbo; Rosenholm, Jessica M; Zhuang, Min; Chen, Zheng-Yi; Chen, Bing; Shu, YilaiMolecular therapy. Methods & clinical development.2020 Sep 11;32953927
Serum Concentrations of Per- and Polyfluoroalkyl Substances and Risk of Renal Cell Carcinoma.Shearer, Joseph J; Callahan, Catherine L; Calafat, Antonia M; Huang, Wen-Yi; Jones, Rena R; Sabbisetti, Venkata S; Freedman, Neal D; Sampson, Joshua N; Silverman, Debra T; Purdue, Mark P; Hofmann, Jonathan NJournal of the National Cancer Institute2021 May 0432944748
Rescuing AAV gene transfer from neutralizing antibodies with an IgG-degrading enzyme.Elmore, Zachary C; Oh, Daniel K; Simon, Katherine E; Fanous, Marco M; Asokan, AravindJCI insight2020 09 1732941184
SARS-CoV-2 infection of human iPSC-derived cardiac cells predicts novel cytopathic features in hearts of COVID-19 patients.Pérez-Bermejo, Juan A; Kang, Serah; Rockwood, Sarah J; Simoneau, Camille R; Joy, David A; Ramadoss, Gokul N; Silva, Ana C; Flanigan, Will R; Li, Huihui; Nakamura, Ken; Whitman, Jeffrey D; Ott, Melanie; Conklin, Bruce R; McDevitt, Todd CbioRxiv : the preprint server for biology.2020 Sep 12;32935097
Rapid, precise quantification of large DNA excisions and inversions by ddPCR.Watry, Hannah L; Feliciano, Carissa M; Gjoni, Ketrin; Takahashi, Gou; Miyaoka, Yuichiro; Conklin, Bruce R; Judge, Luke MScientific reports.2020 09 10;32913194
Adenoviral vectors for in vivo delivery of CRISPR-Cas gene editors.Boucher, Paul; Cui, Xiaoxia; Curiel, David TJournal of controlled release : official journal of the Controlled Release Society2020 11 1032891680
Synthetic immunomodulation with a CRISPR super-repressor in vivo.Moghadam, Farzaneh; LeGraw, Ryan; Velazquez, Jeremy J; Yeo, Nan Cher; Xu, Chenxi; Park, Jin; Chavez, Alejandro; Ebrahimkhani, Mo R; Kiani, SamiraNature cell biology.2020 Sep;32884147
Frame-Hydrogel Methodology for Engineering Highly Functional Cardiac Tissue Constructs.Helfer, Abbigail; Bursac, NenadMethods in molecular biology (Clifton, N.J.).2021;32857373
Opportunities and limitations of genetically modified nonhuman primate models for neuroscience research.Feng, Guoping; Jensen, Frances E; Greely, Henry T; Okano, Hideyuki; Treue, Stefan; Roberts, Angela C; Fox, James G; Caddick, Sarah; Poo, Mu-Ming; Newsome, William T; Morrison, John HProceedings of the National Academy of Sciences of the United States of America.2020 09 29;32817435
The Golgi Calcium ATPase Pump Plays an Essential Role in Adeno-associated Virus Trafficking and Transduction.Madigan, Victoria J; Berry, Garrett E; Tyson, Tyne O; Nardone-White, Dasean; Ark, Jonathan; Elmore, Zachary C; Murlidharan, Giridhar; Vincent, Heather A; Asokan, AravindJournal of virology.2020 Oct 14;32817219
New Directions in Pulmonary Gene Therapy.Vu, Amber; McCray , Jr, Paul BHuman gene therapy2020 0932814451
Treating Cystic Fibrosis with mRNA and CRISPR.Da Silva Sanchez, Alejandro; Paunovska, Kalina; Cristian, Ana; Dahlman, James EHuman gene therapy2020 0932799680
NDAT Targets PI3K-Mediated PD-L1 Upregulation to Reduce Proliferation in Gefitinib-Resistant Colorectal Cancer.Huang, Tung-Yung; Chang, Tung-Cheng; Chin, Yu-Tang; Pan, Yi-Shin; Chang, Wong-Jin; Liu, Feng-Cheng; Hastuti, Ema Dwi; Chiu, Shih-Jiuan; Wang, Shwu-Huey; Changou, Chun A; Li, Zi-Lin; Chen, Yi-Ru; Chu, Hung-Ru; Shih, Ya-Jung; Cheng, R Holland; Wu, AlexanderCells.2020 08 03;32756527
Epigenetic transcriptional reprogramming by WT1 mediates a repair response during podocyte injury.Ettou, Sandrine; Jung, Youngsook L; Miyoshi, Tomoya; Jain, Dhawal; Hiratsuka, Ken; Schumacher, Valerie; Taglienti, Mary E; Morizane, Ryuji; Park, Peter J; Kreidberg, Jordan AScience advances.2020 Jul;32754639
DNA capture by a CRISPR-Cas9-guided adenine base editor.Lapinaite, Audrone; Knott, Gavin J; Palumbo, Cody M; Lin-Shiao, Enrique; Richter, Michelle F; Zhao, Kevin T; Beal, Peter A; Liu, David R; Doudna, Jennifer AScience (New York, N.Y.).2020 07 31;32732424
Advances in microphysiological blood-brain barrier (BBB) models towards drug delivery.Lee, Caleb S; Leong, Kam WCurrent opinion in biotechnology.2020 Dec;32711361
The dawn of non-human primate models for neurodevelopmental disorders.Aida, Tomomi; Feng, GuopingCurrent opinion in genetics & development2020 1232693220
CRISPR-Cas? from huge phages is a hypercompact genome editor.Pausch, Patrick; Al-Shayeb, Basem; Bisom-Rapp, Ezra; Tsuchida, Connor A; Li, Zheng; Cress, Brady F; Knott, Gavin J; Jacobsen, Steven E; Banfield, Jillian F; Doudna, Jennifer AScience (New York, N.Y.).2020 07 17;32675376
Coevolution of Adeno-associated Virus Capsid Antigenicity and Tropism through a Structure-Guided Approach.Havlik, L Patrick; Simon, Katherine E; Smith, J Kennon; Klinc, Kelli A; Tse, Longping V; Oh, Daniel K; Fanous, Marco M; Meganck, Rita M; Mietzsch, Mario; Kleinschmidt, Jürgen; Agbandje-McKenna, Mavis; Asokan, AravindJournal of virology.2020 Sep 15;32669336
Advances in Alpha-1 Antitrypsin Gene Therapy.Lorincz, Reka; Curiel, David TAmerican journal of respiratory cell and molecular biology.2020 Nov;32668173
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
A scoutRNA Is Required for Some Type V CRISPR-Cas Systems.Harrington, Lucas B; Ma, Enbo; Chen, Janice S; Witte, Isaac P; Gertz, Dov; Paez-Espino, David; Al-Shayeb, Basem; Kyrpides, Nikos C; Burstein, David; Banfield, Jillian F; Doudna, Jennifer AMolecular cell.2020 08 06;32645367
A bacterial cytidine deaminase toxin enables CRISPR-free mitochondrial base editing.Mok, Beverly Y; de Moraes, Marcos H; Zeng, Jun; Bosch, Dustin E; Kotrys, Anna V; Raguram, Aditya; Hsu, FoSheng; Radey, Matthew C; Peterson, S Brook; Mootha, Vamsi K; Mougous, Joseph D; Liu, David RNature.2020 07;32641830
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
The delivery challenge: fulfilling the promise of therapeutic genome editing.van Haasteren, Joost; Li, Jie; Scheideler, Olivia J; Murthy, Niren; Schaffer, David VNature biotechnology.2020 07;32601435
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
Induced pluripotent stem cells provide mega insights into kidney disease.Luciani, Alessandro; Freedman, Benjamin SKidney international2020 0732571490
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
Determinants of Base Editing Outcomes from Target Library Analysis and Machine Learning.Arbab, Mandana; Shen, Max W; Mok, Beverly; Wilson, Christopher; Matuszek, ?aneta; Cassa, Christopher A; Liu, David RCell.2020 Jul 23;32533916
Defining Transcription Regulatory Elements in the Human Frataxin Gene: Implications for Gene Therapy.Li, Jixue; Li, Yanjie; Wang, Jun; Gonzalez, Trevor J; Asokan, Aravind; Napierala, Jill S; Napierala, MarekHuman gene therapy2020 0832527155
Combinatorial Library of Cyclic Benzylidene Acetal-Containing pH-Responsive Lipidoid Nanoparticles for Intracellular mRNA Delivery.Li, Yamin; Li, Raissa; Chakraborty, Anirban; Ogurlu, Roza; Zhao, Xuewei; Chen, Jinjin; Xu, QiaobingBioconjugate chemistry.2020 07 15;32520527
In vivo base editing restores sensory transduction and transiently improves auditory function in a mouse model of recessive deafness.Yeh, Wei-Hsi; Shubina-Oleinik, Olga; Levy, Jonathan M; Pan, Bifeng; Newby, Gregory A; Wornow, Michael; Burt, Rachel; Chen, Jonathan C; Holt, Jeffrey R; Liu, David RScience translational medicine2020 06 0332493795
YAP1 withdrawal in hepatoblastoma drives therapeutic differentiation of tumor cells to functional hepatocyte-like cells.Smith, Jordan L; Rodríguez, Tomás C; Mou, Haiwei; Kwan, Suet-Yan; Pratt, Henry; Zhang, Xiao-Ou; Cao, Yueying; Liang, Shunqing; Ozata, Deniz M; Yu, Tianxiong; Yin, Qiangzong; Hazeltine, Max; Weng, Zhiping; Sontheimer, Erik J; Xue, WenHepatology (Baltimore, Md.).2020 May 26;32452550
The developing toolkit of continuous directed evolution.Morrison, Mary S; Podracky, Christopher J; Liu, David RNature chemical biology.2020 Jun;32444838
Protein and mRNA Delivery Enabled by Cholesteryl-Based Biodegradable Lipidoid Nanoparticles.Li, Yamin; Jarvis, Rachel; Zhu, Kuixin; Glass, Zachary; Ogurlu, Roza; Gao, Peiyang; Li, Peixuan; Chen, Jinjin; Yu, Yingjie; Yang, Yongjie; Xu, QiaobingAngewandte Chemie (International ed. in English).2020 May 21;32438474
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
Prime genome editing in rice and wheat.Lin, Qiupeng; Zong, Yuan; Xue, Chenxiao; Wang, Shengxing; Jin, Shuai; Zhu, Zixu; Wang, Yanpeng; Anzalone, Andrew V; Raguram, Aditya; Doman, Jordan L; Liu, David R; Gao, CaixiaNature biotechnology.2020 05;32393904
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
Finding order in slow axonal transport.Roy, SubhojitCurrent opinion in neurobiology.2020 08;32361600
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, RyaNature communications.2020 04 30;32355221
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
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 TJournal of virology2020 05 0432102889
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
The promise and challenge of therapeutic genome editing.Doudna, Jennifer ANature2020 0232051598
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
Noninvasive imaging of renal urea handling by CEST-MRI.Shin, Soo Hyun; Wendland, Michael F; Zhang, Brandon; Tran, An; Tang, Albert; Vandsburger, Moriel HMagnetic resonance in medicine2020 0331483529
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
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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
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