Somatic Cell Genome Editing (318)
Publication TitleAuthorsJournalPublication DatePage NoPubMedID
High-throughput continuous evolution of compact Cas9 variants targeting single-nucleotide-pyrimidine PAMs.Huang, Tony P; Heins, Zachary J; Miller, Shannon M; Wong, Brandon G; Balivada, Pallavi A; Wang, Tina; Khalil, Ahmad S; Liu, David RNature biotechnology2022 Sep 0836076084
Piperazine-derived lipid nanoparticles deliver mRNA to immune cells in vivo.Ni, Huanzhen; Hatit, Marine Z C; Zhao, Kun; Loughrey, David; Lokugamage, Melissa P; Peck, Hannah E; Cid, Ada Del; Muralidharan, Abinaya; Kim, YongTae; Santangelo, Philip J; Dahlman, James ENature communications2022 08 1535970837
Designing and executing prime editing experiments in mammalian cells.Doman, Jordan L; Sousa, Alexander A; Randolph, Peyton B; Chen, Peter J; Liu, David RNature protocols2022 Aug 0835941224
Efficient in vivo base editing via single adeno-associated viruses with size-optimized genomes encoding compact adenine base editors.Davis, Jessie R; Wang, Xiao; Witte, Isaac P; Huang, Tony P; Levy, Jonathan M; Raguram, Aditya; Banskota, Samagya; Seidah, Nabil G; Musunuru, Kiran; Liu, David RNature biomedical engineering2022 Jul 2835902773
Development of 68Ga-Labeled Hepatitis E Virus Nanoparticles for Targeted Drug Delivery and Diagnostics with PET.Lambidis, Elisavet; Chen, Chun-Chieh; Baikoghli, Mo; Imlimthan, Surachet; Khng, You Cheng; Sarparanta, Mirkka; Cheng, R Holland; Airaksinen, Anu JMolecular pharmaceutics2022 08 0135857429
Evaluation Protocol for SARS-CoV-2 Serological Assays.Gededzha, Maemu P; Jugwanth, Sarika; Mampeule, Nakampe; Zwane, Nontobeko; David, Anura; Scott, Lesley; Stevens, Wendy; Mayne, Elizabeth SMethods in molecular biology (Clifton, N.J.)202235838970
SARS-CoV-2 Host Immunogenetic Biomarkers.Gededzha, Maemu P; Mampeule, Nakampe; Gandini, Anastasia; Mayne, Elizabeth SMethods in molecular biology (Clifton, N.J.)202235838957
In situ autofluorescence lifetime assay of a photoreceptor stimulus response in mouse retina and human retinal organoids.Samimi, Kayvan; Pattnaik, Bikash R; Capowski, Elizabeth E; Saha, Krishanu; Gamm, David M; Skala, Melissa CBiomedical optics express2022 Jun 0135781966
Efficient Genome Editing Achieved via Plug-and-Play Adenovirus Piggyback Transport of Cas9/gRNA Complex on Viral Capsid Surface.Lu, Zhi Hong; Li, Jie; Dmitriev, Igor P; Kashentseva, Elena A; Curiel, David TACS nano2022 Jun 2435749339
In vivo engineered B cells secrete high titers of broadly neutralizing anti-HIV antibodies in mice.Nahmad, Alessio D; Lazzarotto, Cicera R; Zelikson, Natalie; Kustin, Talia; Tenuta, Mary; Huang, Deli; Reuveni, Inbal; Nataf, Daniel; Raviv, Yuval; Horovitz-Fried, Miriam; Dotan, Iris; Carmi, Yaron; Rosin-Arbesfeld, Rina; Nemazee, David; Voss, James E; SteNature biotechnology2022 Aug35681059
Inhibition of B-cell lymphoma 2 family proteins alters optical redox ratio, mitochondrial polarization, and cell energetics independent of cell state.Gillette, Amani A; DeStefanis, Rebecca A; Pritzl, Stephanie L; Deming, Dustin A; Skala, Melissa CJournal of biomedical optics2022 0535643815
CRISPR Library Screening in Cultured Cardiomyocytes.DeLuca, Sophia; Bursac, NenadMethods in molecular biology (Clifton, N.J.)202235618895
Label-Free Imaging to Track Reprogramming of Human Somatic Cells.Molugu, Kaivalya; Battistini, Giovanni A; Heaster, Tiffany M; Rouw, Jacob; Guzman, Emmanuel C; Skala, Melissa C; Saha, KrishanuGEN biotechnology2022 Apr 0135586336
A High-Efficiency AAV for Endothelial Cell Transduction Throughout the Central Nervous System.Krolak, Trevor; Chan, Ken Y; Kaplan, Luke; Huang, Qin; Wu, Jason; Zheng, Qingxia; Kozareva, Velina; Beddow, Thomas; Tobey, Isabelle G; Pacouret, Simon; Chen, Albert T; Chan, Yujia A; Ryvkin, Daniel; Gu, Chenghua; Deverman, Benjamin ENature cardiovascular research2022 Apr35571675
The clinical progress of mRNA vaccines and immunotherapies.Barbier, Ann J; Jiang, Allen Yujie; Zhang, Peng; Wooster, Richard; Anderson, Daniel GNature biotechnology2022 0635534554
Prioritization of autoimmune disease-associated genetic variants that perturb regulatory element activity in T cells.Mouri, Kousuke; Guo, Michael H; de Boer, Carl G; Lissner, Michelle M; Harten, Ingrid A; Newby, Gregory A; DeBerg, Hannah A; Platt, Winona F; Gentili, Matteo; Liu, David R; Campbell, Daniel J; Hacohen, Nir; Tewhey, Ryan; Ray, John PNature genetics2022 0535513721
Identification and Validation of CRISPR/Cas9 Off-Target Activity in Hematopoietic Stem and Progenitor Cells.Park, So Hyun; Lee, Ciaran M; Bao, GangMethods in molecular biology (Clifton, N.J.)202235507169
An optimized FusX assembly-based technique to introduce mitochondrial TC-to-TT variations in human cell lines.Kar, Bibekananda; Sabharwal, Ankit; Restrepo-Castillo, Santiago; Simone, Brandon W; Clark, Karl J; Ekker, Stephen CSTAR protocols2022 Jun 1735496789
Modelling ciliopathy phenotypes in human tissues derived from pluripotent stem cells with genetically ablated cilia.Cruz, Nelly M; Reddy, Raghava; McFaline-Figueroa, José L; Tran, Christine; Fu, Hongxia; Freedman, Benjamin SNature biomedical engineering2022 0435478224
Tetrazine-Ligated CRISPR sgRNAs for Efficient Genome Editing.Chen, Zexiang; Devi, Gitali; Arif, Amena; Zamore, Phillip D; Sontheimer, Erik J; Watts, Jonathan KACS chemical biology2022 05 2035446558
Crystal structure of an RNA/DNA strand exchange junction.Cofsky, Joshua C; Knott, Gavin J; Gee, Christine L; Doudna, Jennifer APloS one202235436289
CRISPR-Cas9 bends and twists DNA to read its sequence.Cofsky, Joshua C; Soczek, Katarzyna M; Knott, Gavin J; Nogales, Eva; Doudna, Jennifer ANature structural & molecular biology2022 Apr35422516
Myoblast deactivation within engineered human skeletal muscle creates a transcriptionally heterogeneous population of quiescent satellite-like cells.Wang, Jason; Broer, Torie; Chavez, Taylor; Zhou, Chris J; Tran, Sabrina; Xiang, Yu; Khodabukus, Alastair; Diao, Yarui; Bursac, NenadBiomaterials2022 0535421801
Multivalent designed proteins neutralize SARS-CoV-2 variants of concern and confer protection against infection in mice.Hunt, Andrew C; Case, James Brett; Park, Young-Jun; Cao, Longxing; Wu, Kejia; Walls, Alexandra C; Liu, Zhuoming; Bowen, John E; Yeh, Hsien-Wei; Saini, Shally; Helms, Louisa; Zhao, Yan Ting; Hsiang, Tien-Ying; Starr, Tyler N; Goreshnik, Inna; Kozodoy, Lisa; Carter, Lauren; Ravichandran, Rashmi; Green, Lydia B; Matochko, Wadim L; Thomson, Christy A; Vögeli, Bastian; Krüger, Antje; VanBlargan, Laura A; Chen, Rita E; Ying, Baoling; Bailey, Adam L; Kafai, Natasha M; Boyken, Scott E; Ljubetič, Ajasja; Edman, Natasha; Ueda, George; Chow, Cameron M; Johnson, Max; Addetia, Amin; Navarro, Mary Jane; Panpradist, Nuttada; Gale Jr, Michael; Freedman, Benjamin S; Bloom, Jesse D; Ruohola-Baker, Hannele; Whelan, Sean P J; Stewart, Lance; Diamond, Michael S; Veesler, David; Jewett, Michael C; Baker, DavidScience translational medicine2022 Apr 1235412328
Surface conjugation of antibodies improves nanoparticle uptake in bronchial epithelial cells.Luks, Valerie L; Mandl, Hanna; DiRito, Jenna; Barone, Christina; Freedman-Weiss, Mollie R; Ricciardi, Adele S; Tietjen, Gregory G; Egan, Marie E; Saltzman, W Mark; Stitelman, David HPloS one202235385514
In vivo base editing rescues cone photoreceptors in a mouse model of early-onset inherited retinal degeneration.Choi, Elliot H; Suh, Susie; Foik, Andrzej T; Leinonen, Henri; Newby, Gregory A; Gao, Xin D; Banskota, Samagya; Hoang, Thanh; Du, Samuel W; Dong, Zhiqian; Raguram, Aditya; Kohli, Sajeev; Blackshaw, Seth; Lyon, David C; Liu, David R; Palczewski, KrzysztofNature communications2022 Apr 0535383196
CRISPR-free base editors with enhanced activity and expanded targeting scope in mitochondrial and nuclear DNA.Mok, Beverly Y; Kotrys, Anna V; Raguram, Aditya; Huang, Tony P; Mootha, Vamsi K; Liu, David RNature biotechnology2022 Apr 0435379961
Physiology assays in human kidney organoids.Freedman, Benjamin SAmerican journal of physiology. Renal physiology2022 06 0135379001
Efficient targeted transgenesis of large donor DNA into multiple mouse genetic backgrounds using bacteriophage Bxb1 integrase.Low, Benjamin E; Hosur, Vishnu; Lesbirel, Simon; Wiles, Michael VScientific reports2022 03 3135361849
Robust, Durable Gene Activation In Vivo via mRNA-Encoded Activators.Beyersdorf, Jared P; Bawage, Swapnil; Iglesias, Nahid; Peck, Hannah E; Hobbs, Ryan A; Wroe, Jay A; Zurla, Chiara; Gersbach, Charles A; Santangelo, Philip JACS nano2022 Mar 3135357116
Impact of baseline culture conditions of cancer organoids when determining therapeutic response and tumor heterogeneity.DeStefanis, Rebecca A; Kratz, Jeremy D; Olson, Autumn M; Sunil, Aishwarya; DeZeeuw, Alyssa K; Gillette, Amani A; Sha, Gioia C; Johnson, Katherine A; Pasch, Cheri A; Clipson, Linda; Skala, Melissa C; Deming, Dustin AScientific reports2022 03 2535338174
AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice.Wang, Jiaming; Zhang, Yue; Mendonca, Craig A; Yukselen, Onur; Muneeruddin, Khaja; Ren, Lingzhi; Liang, Jialing; Zhou, Chen; Xie, Jun; Li, Jia; Jiang, Zhong; Kucukural, Alper; Shaffer, Scott A; Gao, Guangping; Wang, DanNature2022 0435322228
Human and Insect Cell-Produced Recombinant Adeno-Associated Viruses Show Differences in Genome Heterogeneity.Tran, Ngoc Tam; Lecomte, Emilie; Saleun, Sylvie; Namkung, Suk; Robin, Cécile; Weber, Kristina; Devine, Eric; Blouin, Veronique; Adjali, Oumeya; Ayuso, Eduard; Gao, Guangping; Penaud-Budloo, Magalie; Tai, Phillip W LHuman gene therapy2022 0435293222
Translational Utility of the Nonhuman Primate Model.Tarantal, Alice F; Hartigan-O'Connor, Dennis J; Noctor, Stephen CBiological psychiatry. Cognitive neuroscience and neuroimaging2022 0535283343
Durability of transgene expression after rAAV gene therapy.Muhuri, Manish; Levy, Daniel I; Schulz, Martin; McCarty, Douglas; Gao, GuangpingMolecular therapy : the journal of the American Society of Gene Therapy2022 04 0635283274
Technologies to Assess Drug Response and Heterogeneity in Patient-Derived Cancer Organoids.Skala, Melissa C; Deming, Dustin A; Kratz, Jeremy DAnnual review of biomedical engineering2022 Feb 0835259932
Design of therapeutic biomaterials to control inflammation.Tu, Zhaoxu; Zhong, Yiling; Hu, Hanze; Shao, Dan; Haag, Rainer; Schirner, Michael; Lee, Jaewoo; Sullenger, Bruce; Leong, Kam WNature reviews. Materials2022 Feb 2835251702
Clinical magnetic hyperthermia requires integrated magnetic particle imaging.Healy, Sean; Bakuzis, Andris F; Goodwill, Patrick W; Attaluri, Anilchandra; Bulte, Jeff W M; Ivkov, RobertWiley interdisciplinary reviews. Nanomedicine and nanobiotechnology2022 May35238181
In Vivo Gene Editing in Lipid and Atherosclerosis Research.De Giorgi, Marco; Jarrett, Kelsey E; de Aguiar Vallim, Thomas Q; Lagor, William RMethods in molecular biology (Clifton, N.J.)202235237996
Antispacer peptide nucleic acids for sequence-specific CRISPR-Cas9 modulation.Economos, Nicholas G; Quijano, Elias; Carufe, Kelly E W; Perera, J Dinithi R; Glazer, Peter MNucleic acids research2022 Mar 0335235944
Modeling injury and repair in kidney organoids reveals that homologous recombination governs tubular intrinsic repair.Gupta, Navin; Matsumoto, Takuya; Hiratsuka, Ken; Garcia Saiz, Edgar; Galichon, Pierre; Miyoshi, Tomoya; Susa, Koichiro; Tatsumoto, Narihito; Yamashita, Michifumi; Morizane, RyujiScience translational medicine2022 Mar 0235235339
Modeling genetic diseases in nonhuman primates through embryonic and germline modification: Considerations and challenges.Schmidt, Jenna K; Jones, Kathryn M; Van Vleck, Trevor; Emborg, Marina EScience translational medicine2022 03 0235235338
Treating Cardiovascular Disease with Liver Genome Engineering.Hurley, Ayrea; Lagor, William RCurrent atherosclerosis reports2022 Feb35230602
Magnetic Iron Oxide Nanoparticles for Biomedical Applications.Jiang, Kaiyi; Zhang, Linlin; Bao, GangCurrent opinion in biomedical engineering2021 Dec35211662
Rapid, accurate mapping of transgene integration in viable rhesus macaque embryos using enhanced-specificity tagmentation-assisted PCR.Ryu, Junghyun; Chan, William; Wettengel, Jochen M; Hanna, Carol B; Burwitz, Benjamin J; Hennebold, Jon D; Bimber, Benjamin NMolecular therapy. Methods & clinical development2022 Mar 1035211637
Transcription factor protein interactomes reveal genetic determinants in heart disease.Gonzalez-Teran, Barbara; Pittman, Maureen; Felix, Franco; Thomas, Reuben; Richmond-Buccola, Desmond; Hüttenhain, Ruth; Choudhary, Krishna; Moroni, Elisabetta; Costa, Mauro W; Huang, Yu; Padmanabhan, Arun; Alexanian, Michael; Lee, Clara Youngna; Maven, Bonnie E J; Samse-Knapp, Kaitlen; Morton, Sarah U; McGregor, Michael; Gifford, Casey A; Seidman, J G; Seidman, Christine E; Gelb, Bruce D; Colombo, Giorgio; Conklin, Bruce R; Black, Brian L; Bruneau, Benoit G; Krogan, Nevan J; Pollard, Katherine S; Srivastava, DeepakCell2022 03 0335182466
Derivation of extra-embryonic and intra-embryonic macrophage lineages from human pluripotent stem cells.Bredemeyer, Andrea L; Amrute, Junedh M; Koenig, Andrew L; Idol, Rachel A; He, Li; Luff, Stephanie A; Dege, Carissa; Leid, Jamison M; Schilling, Joel D; Hinson, J Travis; Dinauer, Mary C; Sturgeon, Christopher M; Lavine, Kory JDevelopment (Cambridge, England)2022 04 1535178561
Lung-selective mRNA delivery of synthetic lipid nanoparticles for the treatment of pulmonary lymphangioleiomyomatosis.Qiu, Min; Tang, Yan; Chen, Jinjin; Muriph, Rachel; Ye, Zhongfeng; Huang, Changfeng; Evans, Jason; Henske, Elizabeth P; Xu, QiaobingProceedings of the National Academy of Sciences of the United States of America2022 02 2235173043
A self-immolative linker that releases thiols detects penicillin amidase and nitroreductase with high sensitivity via absorption spectroscopy.Espinoza, Eli M; Røise, Joachim J; He, Maomao; Li, I-Che; Agatep, Alvin K; Udenyi, Patrick; Han, Hesong; Jackson, Nicole; Kerr, D Lucas; Chen, Dake; Stentzel, Michael R; Ruan, Emily; Riley, Lee; Murthy, NirenChemical communications (Cambridge, England)2022 Mar 0335170593
Nonhuman Primates in Translational Research.Tarantal, Alice F; Noctor, Stephen C; Hartigan-O'Connor, Dennis JAnnual review of animal biosciences2022 02 1535167321
A Human-derived Dual MRI/PET Reporter Gene System with High Translational Potential for Cell Tracking.Shalaby, Nourhan; Kelly, John; Martinez, Francisco; Fox, Mathew; Qi, Qi; Thiessen, Jonathan; Hicks, Justin; Scholl, Timothy J; Ronald, John AMolecular imaging and biology2022 0435146614
Species-dependent in vivo mRNA delivery and cellular responses to nanoparticles.Hatit, Marine Z C; Lokugamage, Melissa P; Dobrowolski, Curtis N; Paunovska, Kalina; Ni, Huanzhen; Zhao, Kun; Vanover, Daryll; Beyersdorf, Jared; Peck, Hannah E; Loughrey, David; Sato, Manaka; Cristian, Ana; Santangelo, Philip J; Dahlman, James ENature nanotechnology2022 Mar35132167
pH-Responsive Polymer Nanoparticles for Efficient Delivery of Cas9 Ribonucleoprotein With or Without Donor DNA.Xie, Ruosen; Wang, Xiuxiu; Wang, Yuyuan; Ye, Mingzhou; Zhao, Yi; Yandell, Brian S; Gong, ShaoqinAdvanced materials (Deerfield Beach, Fla.)2022 Feb 0435119139
Genome-wide detection of CRISPR editing in vivo using GUIDE-tag.Liang, Shun-Qing; Liu, Pengpeng; Smith, Jordan L; Mintzer, Esther; Maitland, Stacy; Dong, Xiaolong; Yang, Qiyuan; Lee, Jonathan; Haynes, Cole M; Zhu, Lihua Julie; Watts, Jonathan K; Sontheimer, Erik J; Wolfe, Scot A; Xue, WenNature communications2022 01 2135064134
Detecting Cardiovascular Protein-Protein Interactions by Proximity Proteomics.Circulation research2022 01 2135050691
BRD2 inhibition blocks SARS-CoV-2 infection by reducing transcription of the host cell receptor ACE2.Samelson, Avi J; Tran, Quang Dinh; Robinot, Rémy; Carrau, Lucia; Rezelj, Veronica V; Kain, Alice Mac; Chen, Merissa; Ramadoss, Gokul N; Guo, Xiaoyan; Lim, Shion A; Lui, Irene; Nuñez, James K; Rockwood, Sarah J; Wang, Jianhui; Liu, Na; Carlson-Stevermer, Jared; Oki, Jennifer; Maures, Travis; Holden, Kevin; Weissman, Jonathan S; Wells, James A; Conklin, Bruce R; TenOever, Benjamin R; Chakrabarti, Lisa A; Vignuzzi, Marco; Tian, Ruilin; Kampmann, MartinNature cell biology2022 0135027731
Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins.Banskota, Samagya; Raguram, Aditya; Suh, Susie; Du, Samuel W; Davis, Jessie R; Choi, Elliot H; Wang, Xiao; Nielsen, Sarah C; Newby, Gregory A; Randolph, Peyton B; Osborn, Mark J; Musunuru, Kiran; Palczewski, Krzysztof; Liu, David RCell2022 01 2035021064
A flexible split prime editor using truncated reverse transcriptase improves dual-AAV delivery in mouse liver.Zheng, Chunwei; Liang, Shun-Qing; Liu, Bin; Liu, Pengpeng; Kwan, Suet-Yan; Wolfe, Scot A; Xue, WenMolecular therapy : the journal of the American Society of Gene Therapy2022 Jan 0534998953
Editorial: "AAV Gene Therapy: Immunology and Immunotherapeutics".Martinez-Navio, José M; Paulk, Nicole K; Gao, GuangpingFrontiers in immunology202134992614
rAAV-delivered PTEN therapeutics for prostate cancer.Ai, Jianzhong; Li, Jia; Su, Qin; Ma, Hong; Wei, Qiang; Li, Hong; Gao, GuangpingMolecular therapy. Nucleic acids2022 Mar 0834976432
CRISPR/Cas9 ribonucleoprotein-mediated genome and epigenome editing in mammalian cells.Advanced drug delivery reviews2022 Feb34942274
Acid-Sensitive Surfactants Enhance the Delivery of Nucleic Acids.Røise, Joachim Justad; Han, Hesong; Li, Jie; Kerr, D Lucas; Taing, Chung; Behrouzi, Kamyar; He, Maomao; Ruan, Emily; Chan, Lienna Y; Espinoza, Eli M; Reinhard, Sören; Thakker, Kanav; Kwon, Justin; Mofrad, Mohammad R K; Murthy, NirenMolecular pharmaceutics2022 01 0334931518
Reading Frame Repair of TTN Truncation Variants Restores Titin Quantity and Functions.Romano, Robert; Ghahremani, Shahnaz; Zimmerman, Talia; Legere, Nicholas; Thakar, Ketan; Ladha, Feria A; Pettinato, Anthony M; Hinson, J TravisCirculation2022 01 1834905694
Breast cancer immunotherapy: current biomarkers and the potential of in vitro assays.Skala, Melissa C; Ayuso, Jose M; Burkard, Mark E; Deming, Dustin ACurrent opinion in biomedical engineering2021 Mar34901585
AAV capsid variants with brain-wide transgene expression and decreased liver targeting after intravenous delivery in mouse and marmoset.Goertsen, David; Flytzanis, Nicholas C; Goeden, Nick; Chuapoco, Miguel R; Cummins, Alexander; Chen, Yijing; Fan, Yingying; Zhang, Qiangge; Sharma, Jitendra; Duan, Yangyang; Wang, Liping; Feng, Guoping; Chen, Yu; Ip, Nancy Y; Pickel, James; Gradinaru, VivianaNature neuroscience2022 0134887588
Programmable deletion, replacement, integration and inversion of large DNA sequences with twin prime editing.Anzalone, Andrew V; Gao, Xin D; Podracky, Christopher J; Nelson, Andrew T; Koblan, Luke W; Raguram, Aditya; Levy, Jonathan M; Mercer, Jaron A M; Liu, David RNature biotechnology2021 Dec 0934887556
Author Correction: Engineered pegRNAs improve prime editing efficiency.Nelson, James W; Randolph, Peyton B; Shen, Simon P; Everette, Kelcee A; Chen, Peter J; Anzalone, Andrew V; An, Meirui; Newby, Gregory A; Chen, Jonathan C; Hsu, Alvin; Liu, David RNature biotechnology2021 Dec 0834880468
The Coiled-Coil Forming Peptide (KVSALKE)5 Is a Cell Penetrating Peptide that Enhances the Intracellular Delivery of Proteins.Li, Jie; Tuma, Jan; Han, Hesong; Kim, Hansol; Wilson, Ross C; Lee, Hye Young; Murthy, NirenAdvanced healthcare materials2021 Dec 0334861744
The FusX TALE Base Editor (FusXTBE) for Rapid Mitochondrial DNA Programming of Human Cells In Vitro and Zebrafish Disease Models In Vivo.Sabharwal, Ankit; Kar, Bibekananda; Restrepo-Castillo, Santiago; Holmberg, Shannon R; Mathew, Neal D; Kendall, Benjamin Luke; Cotter, Ryan P; WareJoncas, Zachary; Seiler, Christoph; Nakamaru-Ogiso, Eiko; Clark, Karl J; Ekker, Stephen CThe CRISPR journal2021 1234847747
CRISPR-enhanced human adipocyte browning as cell therapy for metabolic disease.Tsagkaraki, Emmanouela; Nicoloro, Sarah M; DeSouza, Tiffany; Solivan-Rivera, Javier; Desai, Anand; Lifshitz, Lawrence M; Shen, Yuefei; Kelly, Mark; Guilherme, Adilson; Henriques, Felipe; Amrani, Nadia; Ibraheim, Raed; Rodriguez, Tomas C; Luk, Kevin; Maitland, Stacy; Friedline, Randall H; Tauer, Lauren; Hu, Xiaodi; Kim, Jason K; Wolfe, Scot A; Sontheimer, Erik J; Corvera, Silvia; Czech, Michael PNature communications2021 11 2634836963
Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models.Hakim, Chady H; Kumar, Sandeep R P; Pérez-López, Dennis O; Wasala, Nalinda B; Zhang, Dong; Yue, Yongping; Teixeira, James; Pan, Xiufang; Zhang, Keqing; Million, Emily D; Nelson, Christopher E; Metzger, Samantha; Han, Jin; Louderman, Jacqueline A; Schmidt, Florian; Feng, Feng; Grimm, Dirk; Smith, Bruce F; Yao, Gang; Yang, N Nora; Gersbach, Charles A; Chen, Shi-Jie; Herzog, Roland W; Duan, DongshengNature communications2021 11 2434819506
Deleting DNMT3A in CAR T cells prevents exhaustion and enhances antitumor activity.Prinzing, Brooke; Zebley, Caitlin C; Petersen, Christopher T; Fan, Yiping; Anido, Alejandro Allo; Yi, Zhongzhen; Nguyen, Phuong; Houke, Haley; Bell, Matthew; Haydar, Dalia; Brown, Charmaine; Boi, Shannon K; Alli, Shanta; Crawford, Jeremy Chase; Riberdy, Janice M; Park, Jeoungeun J; Zhou, Sheng; Velasquez, Mireya Paulina; DeRenzo, Chris; Lazzarotto, Cicera R; Tsai, Shengdar Q; Vogel, Peter; Pruett-Miller, Shondra M; Langfitt, Deanna M; Gottschalk, Stephen; Youngblood, Ben; Krenciute, GiedreScience translational medicine2021 Nov 1734788079
Synthetic biology: at the crossroads of genetic engineering and human therapeutics-a Keystone Symposia report.Cable, Jennifer; Leonard, Joshua N; Lu, Timothy K; Xie, Zhen; Chang, Matthew Wook; Fernández, Luis Ángel; Lora, José M; Kaufman, Howard L; Quintana, Francisco J; Geiger, Roger; F Lesser, Cammie; Lynch, Jason P; Hava, David L; Cornish, Virginia W; Lee, Gary K; DiAndreth, Breanna; Fero, Michael; Srivastava, Rajkamal; De Coster, Tim; Roybal, Kole T; Rackham, Owen J L; Kiani, Samira; Zhu, Iowis; Hernandez-Lopez, Rogelio A; Guo, Tingxi; Chen, William C WAnnals of the New York Academy of Sciences2021 1234786712
Neuromuscular Development and Disease: Learning From in vitro and in vivo Models.Fralish, Zachary; Lotz, Ethan M; Chavez, Taylor; Khodabukus, Alastair; Bursac, NenadFrontiers in cell and developmental biology202134778273
Defining genome-wide CRISPR-Cas genome-editing nuclease activity with GUIDE-seq.Malinin, Nikolay L; Lee, GaHyun; Lazzarotto, Cicera R; Li, Yichao; Zheng, Zongli; Nguyen, Nhu T; Liebers, Matthew; Topkar, Ved V; Iafrate, A John; Le, Long P; Aryee, Martin J; Joung, J Keith; Tsai, Shengdar QNature protocols2021 Nov 1234773119
Cross-validation of SARS-CoV-2 responses in kidney organoids and clinical populations.Helms, Louisa; Marchiano, Silvia; Stanaway, Ian B; Hsiang, Tien-Ying; Juliar, Benjamin A; Saini, Shally; Zhao, Yan Ting; Khanna, Akshita; Menon, Rajasree; Alakwaa, Fadhl; Mikacenic, Carmen; Morrell, Eric D; Wurfel, Mark M; Kretzler, Matthias; Harder, Jennifer L; Murry, Charles E; Himmelfarb, Jonathan; Ruohola-Baker, Hannele; Bhatraju, Pavan K; Gale Jr, Michael; Freedman, Benjamin SJCI insight2021 12 2234767537
Disruption of HIV-1 co-receptors CCR5 and CXCR4 in primary human T cells and hematopoietic stem and progenitor cells using base editing.Knipping, Friederike; Newby, Gregory A; Eide, Cindy R; McElroy, Amber N; Nielsen, Sarah C; Smith, Kyle; Fang, Yongxing; Cornu, Tatjana I; Costa, Caroline; Gutierrez-Guerrero, Alejandra; Bingea, Samuel P; Feser, Colby J; Steinbeck, Benjamin; Hippen, Keli L; Blazar, Bruce R; McCaffrey, Anton; Mussolino, Claudio; Verhoeyen, Els; Tolar, Jakub; Liu, David R; Osborn, Mark JMolecular therapy : the journal of the American Society of Gene Therapy2022 01 0534737067
Gain-of-function cardiomyopathic mutations in RBM20 rewire splicing regulation and re-distribute ribonucleoprotein granules within processing bodies.Fenix, Aidan M; Miyaoka, Yuichiro; Bertero, Alessandro; Blue, Steven M; Spindler, Matthew J; Tan, Kenneth K B; Perez-Bermejo, Juan A; Chan, Amanda H; Mayerl, Steven J; Nguyen, Trieu D; Russell, Caitlin R; Lizarraga, Paweena P; Truong, Annie; So, Po-Lin; Kulkarni, Aishwarya; Chetal, Kashish; Sathe, Shashank; Sniadecki, Nathan J; Yeo, Gene W; Murry, Charles E; Conklin, Bruce R; Salomonis, NathanNature communications2021 11 0334732726
Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo.Ibraheim, Raed; Tai, Phillip W L; Mir, Aamir; Javeed, Nida; Wang, Jiaming; Rodríguez, Tomás C; Namkung, Suk; Nelson, Samantha; Khokhar, Eraj Shafiq; Mintzer, Esther; Maitland, Stacy; Chen, Zexiang; Cao, Yueying; Tsagkaraki, Emmanouela; Wolfe, Scot A; Wang, Dan; Pai, Athma A; Xue, Wen; Gao, Guangping; Sontheimer, Erik JNature communications2021 11 0134725353
AAV5 delivery of CRISPR-Cas9 supports effective genome editing in mouse lung airway.Liang, Shun-Qing; Walkey, Christopher J; Martinez, Alexa E; Su, Qin; Dickinson, Mary E; Wang, Dan; Lagor, William R; Heaney, Jason D; Gao, Guangping; Xue, WenMolecular therapy : the journal of the American Society of Gene Therapy2021 Oct 2334695545
Developing Biodegradable Lipid Nanoparticles for Intracellular mRNA Delivery and Genome Editing.Qiu, Min; Li, Yamin; Bloomer, Hanan; Xu, QiaobingAccounts of chemical research2021 11 0234668716
5'-Modifications improve potency and efficacy of DNA donors for precision genome editing.Ghanta, Krishna S; Chen, Zexiang; Mir, Aamir; Dokshin, Gregoriy A; Krishnamurthy, Pranathi M; Yoon, Yeonsoo; Gallant, Judith; Xu, Ping; Zhang, Xiao-Ou; Ozturk, Ahmet Rasit; Shin, Masahiro; Idrizi, Feston; Liu, Pengpeng; Gneid, Hassan; Edraki, Alireza; Lawson, Nathan D; Rivera-Pérez, Jaime A; Sontheimer, Erik J; Watts, Jonathan K; Mello, Craig CeLife2021 10 1934665130
SnRNA sequencing defines signaling by RBC-derived extracellular vesicles in the murine heart.Valkov, Nedyalka; Das, Avash; Tucker, Nathan R; Li, Guoping; Salvador, Ane M; Chaffin, Mark D; Pereira De Oliveira Junior, Getulio; Kur, Ivan; Gokulnath, Priyanka; Ziegler, Olivia; Yeri, Ashish; Lu, Shulin; Khamesra, Aushee; Xiao, Chunyang; Rodosthenous, Life science alliance2021 Dec34663679
Enhanced prime editing systems by manipulating cellular determinants of editing outcomes.Chen, Peter J; Hussmann, Jeffrey A; Yan, Jun; Knipping, Friederike; Ravisankar, Purnima; Chen, Pin-Fang; Chen, Cidi; Nelson, James W; Newby, Gregory A; Sahin, Mustafa; Osborn, Mark J; Weissman, Jonathan S; Adamson, Britt; Liu, David RCell2021 Oct 2834653350
Deletion and replacement of long genomic sequences using prime editing.Jiang, Tingting; Zhang, Xiao-Ou; Weng, Zhiping; Xue, WenNature biotechnology2021 Oct 1434650270
Disulfide-compatible phage-assisted continuous evolution in the periplasmic space.Morrison, Mary S; Wang, Tina; Raguram, Aditya; Hemez, Colin; Liu, David RNature communications2021 10 1334645844
Surface Topography of Polyethylene Glycol Shell Nanoparticles Formed from Bottlebrush Block Copolymers Controls Interactions with Proteins and Cells.Grundler, Julian; Shin, Kwangsoo; Suh, Hee-Won; Zhong, Mingjiang; Saltzman, W MarkACS nano2021 10 2634633171
Kidney development to kidney organoids and back again.Gupta, Navin; Morizane, RyujiSeminars in cell & developmental biology2021 Oct 0634627669
Optimization of lipid nanoparticles for the delivery of nebulized therapeutic mRNA to the lungs.Lokugamage, Melissa P; Vanover, Daryll; Beyersdorf, Jared; Hatit, Marine Z C; Rotolo, Laura; Echeverri, Elisa Schrader; Peck, Hannah E; Ni, Huanzhen; Yoon, Jeong-Kee; Kim, YongTae; Santangelo, Philip J; Dahlman, James ENature biomedical engineering2021 0934616046
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
Functional correction of CFTR mutations in human airway epithelial cells using adenine base editors.Krishnamurthy, Sateesh; Traore, Soumba; Cooney, Ashley L; Brommel, Christian M; Kulhankova, Katarina; Sinn, Patrick L; Newby, Gregory A; Liu, David R; McCray, Paul BNucleic acids research2021 Oct 1134520545
In vivo somatic cell base editing and prime editing.Newby, Gregory A; Liu, David RMolecular therapy : the journal of the American Society of Gene Therapy2021 Nov 0334509669
Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy.Pickar-Oliver, Adrian; Gough, Veronica; Bohning, Joel D; Liu, Siyan; Robinson-Hamm, Jacqueline N; Daniels, Heather; Majoros, William H; Devlin, Garth; Asokan, Aravind; Gersbach, Charles AMolecular therapy : the journal of the American Society of Gene Therapy2021 Nov 0334509668
Allele-Specific Gene Editing Rescues Pathology in a Human Model of Charcot-Marie-Tooth Disease Type 2E.Feliciano, Carissa M; Wu, Kenneth; Watry, Hannah L; Marley, Chiara B E; Ramadoss, Gokul N; Ghanim, Hana Y; Liu, Angela Z; Zholudeva, Lyandysha V; McDevitt, Todd C; Saporta, Mario A; Conklin, Bruce R; Judge, Luke MFrontiers in cell and developmental biology202134485306
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
rAAV-based and intraprostatically delivered miR-34a therapeutics for efficient inhibition of prostate cancer progression.Ai, Jianzhong; Li, Jia; Su, Qin; Ma, Hong; He, Ran; Wei, Qiang; Li, Hong; Gao, GuangpingGene therapy2021 Jul 0634226687
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
Prediction and validation of hematopoietic stem and progenitor cell off-target editing in transplanted rhesus macaques.AlJanahi, Aisha A; Lazzarotto, Cicera R; Chen, Shirley; Shin, Tae-Hoon; Cordes, Stefan; Fan, Xing; Jabara, Isabel; Zhou, Yifan; Young, David J; Lee, Byung-Chul; Yu, Kyung-Rok; Li, Yuesheng; Toms, Bradley; Tunc, Ilker; Hong, So Gun; Truitt, Lauren L; Klermund, Julia; Andrieux, Geoffroy; Kim, Miriam Y; Cathomen, Toni; Gill, Saar; Tsai, Shengdar Q; Dunbar, Cynthia EMolecular therapy : the journal of the American Society of Gene Therapy2022 01 0534174439
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
Magnetic Forces Enable Control of Biological Processes In Vivo.Bao, GangJournal of applied mechanics2021 Mar 0134168385
Targeting the Apoa1 locus for liver-directed gene therapy.De Giorgi, Marco; Li, Ang; Hurley, Ayrea; Barzi, Mercedes; Doerfler, Alexandria M; Cherayil, Nikitha A; Smith, Harrison E; Brown, Jonathan D; Lin, Charles Y; Bissig, Karl-Dimiter; Bao, Gang; Lagor, William RMolecular therapy. Methods & clinical development2021 Jun 1134141821
Mechanisms of angiogenic incompetence in Hutchinson-Gilford progeria via downregulation of endothelial NOS.Gete, Yantenew G; Koblan, Luke W; Mao, Xiaojing; Trappio, Mason; Mahadik, Bhushan; Fisher, John P; Liu, David R; Cao, KanAging cell2021 0734086398
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
3D cell culture models: Drug pharmacokinetics, safety assessment, and regulatory consideration.Wang, Hongbing; Brown, Paul C; Chow, Edwin C Y; Ewart, Lorna; Ferguson, Stephen S; Fitzpatrick, Suzanne; Freedman, Benjamin S; Guo, Grace L; Hedrich, William; Heyward, Scott; Hickman, James; Isoherranen, Nina; Li, Albert P; Liu, Qi; Mumenthaler, Shannon M; Polli, James; Proctor, William R; Ribeiro, Alexandre; Wang, Jian-Ying; Wange, Ronald L; Huang, Shiew-MeiClinical and translational science2021 0933982436
Three-dimensional tissue-engineered human skeletal muscle model of Pompe disease.Wang, Jason; Zhou, Chris J; Khodabukus, Alastair; Tran, Sabrina; Han, Sang-Oh; Carlson, Aaron L; Madden, Lauran; Kishnani, Priya S; Koeberl, Dwight D; Bursac, NenadCommunications biology2021 05 0533953320
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
AAV-Mediated Gene Therapy for Glycosphingolipid Biosynthesis Deficiencies.Yang, Huiya; Brown Jr, Robert H; Wang, Dan; Strauss, Kevin A; Gao, GuangpingTrends in molecular medicine2021 0633714697
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
Redirecting Vesicular Transport to Improve Nonviral Delivery of Molecular Cargo.Mao, Mao; Chang, Chun-Chi; Pickar-Oliver, Adrian; Cervia, Lisa D; Wang, Liangli; Ji, Jing; Liton, Paloma B; Gersbach, Charles A; Yuan, FanAdvanced biosystems2020 0833179869
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
Reactions to the National Academies/Royal Society Report on Heritable Human Genome Editing.Angrist, Misha; Barrangou, Rodolphe; Baylis, Françoise; Brokowski, Carolyn; Burgio, Gaetan; Caplan, Arthur; Chapman, Carolyn Riley; Church, George M; Cook-Deegan, Robert; Cwik, Bryan; Doudna, Jennifer A; Evans, John H; Greely, Henry T; Hercher, Laura; Hurlbut, J Benjamin; Hynes, Richard O; Ishii, Tetsuya; Kiani, Samira; Lee, LaTasha Hoskins; Levrier, Guillaume; Liu, David R; Lunshof, Jeantine E; Macintosh, Kerry Lynn; Mathews, Debra J H; Meslin, Eric M; Mills, Peter H R; Montoliu, Lluis; Musunuru, Kiran; Nicol, Dianne; O'Neill, Helen; Qiu, Renzong; Ranisch, Robert; Sherkow, Jacob S; Soni, Sheetal; Terry, Sharon; Topol, Eric; Williamson, Robert; Zhang, Feng; Davies, KevinThe CRISPR journal2020 1033095048
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
Neurotransmitter-derived lipidoids (NT-lipidoids) for enhanced brain delivery through intravenous injection.Ma, Feihe; Yang, Liu; Sun, Zhuorui; Chen, Jinjin; Rui, Xuehui; Glass, Zachary; Xu, QiaobingScience advances2020 0732832671
Single C-to-T substitution using engineered APOBEC3G-nCas9 base editors with minimum genome- and transcriptome-wide off-target effects.Lee, Sangsin; Ding, Ning; Sun, Yidi; Yuan, Tanglong; Li, Jing; Yuan, Qichen; Liu, Lizhong; Yang, Jie; Wang, Qian; Kolomeisky, Anatoly B; Hilton, Isaac B; Zuo, Erwei; Gao, XueScience advances2020 0732832622
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
mRNA Delivery Using Bioreducible Lipidoid Nanoparticles Facilitates Neural Differentiation of Human Mesenchymal Stem Cells.Zhao, Xuewei; Glass, Zachary; Chen, Jinjin; Yang, Liu; Kaplan, David L; Xu, QiaobingAdvanced healthcare materials2021 0232815325
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
Imidazole-Based Synthetic Lipidoids for In Vivo mRNA Delivery into Primary T Lymphocytes.Zhao, Xuewei; Chen, Jinjin; Qiu, Min; Li, Yamin; Glass, Zachary; Xu, QiaobingAngewandte Chemie (International ed. in English)2020 11 0232662132
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
Transcriptomic and Proteostasis Networks of CFTR and the Development of Small Molecule Modulators for the Treatment of Cystic Fibrosis Lung Disease.Strub, Matthew D; McCray Jr, Paul BGenes2020 05 1332414011
Effect of luminal flow on doming of mpkCCD cells in a 3D perfusable kidney cortical collecting duct model.Rein, Joshua L; Heja, Szilvia; Flores, Daniel; Carrisoza-Gaytán, Rolando; Lin, Neil Y C; Homan, Kimberly A; Lewis, Jennifer A; Satlin, Lisa MAmerican journal of physiology. Cell physiology2020 07 0132401606
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
CRISPR genome editing in stem cells turns to gold.Asokan, AravindNature materials2019 1031537943
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
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
Kidney organoids in translational medicine: Disease modeling and regenerative medicine.Miyoshi, Tomoya; Hiratsuka, Ken; Saiz, Edgar Garcia; Morizane, RyujiDevelopmental dynamics : an official publication of the American Association of Anatomists2020 0130843293
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