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The search results on this publication page are automated on a monthly schedule based on acknowledgment of NIH Common Fund award numbers and intramural awards. Therefore, this list is not an exhaustive or error-free account of the program’s publications.
Somatic Cell Genome Editing (371)
| Publication Title | Authors | Journal | Publication Date | Page No | PubMedID |
|---|---|---|---|---|---|
| Mitochondrial Base Editing: Recent Advances towards Therapeutic Opportunities. | Kar, Bibekananda; Castillo, Santiago R; Sabharwal, Ankit; Clark, Karl J; Ekker, Stephen C | International journal of molecular sciences | 2023 Mar 18 | 36982871 | |
| A parathyroid hormone/salt-inducible kinase signaling axis controls renal vitamin D activation and organismal calcium homeostasis. | Yoon, Sung-Hee; Meyer, Mark B; Arevalo Rivas, Carlos; Tekguc, Murat; Zhang, Chengcheng; Wang, Jialiang S; Castro Andrade, Christian D; Strauss, Katelyn E; Sato, Tadatoshi; Benkusky, Nancy; Lee, Seong Min; Berdeaux, Rebecca; Foretz, Marc; Sundberg, Thomas B; Xavier, Ramnik J; Adelmann, Charles H; Brooks, Daniel J; Anselmo, Anthony; Sadreyev, Ruslan I; Rosales, Ivy A; Fisher, David E; Gupta, Navin; Morizane, Ryuji; Greka, Anna; Pike, J Wesley; Mannstadt, Michael; Wein, Marc N | The Journal of clinical investigation | 2023 Mar 02 | 36862513 | |
| Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in mice. | Reichart, Daniel; Newby, Gregory A; Wakimoto, Hiroko; Lun, Mingyue; Gorham, Joshua M; Curran, Justin J; Raguram, Aditya; DeLaughter, Daniel M; Conner, David A; Marsiglia, Júlia D C; Kohli, Sajeev; Chmatal, Lukas; Page, David C; Zabaleta, Nerea; Vandenberghe, Luk; Liu, David R; Seidman, Jonathan G; Seidman, Christine | Nature medicine | 2023 Feb | 412-421 | 36797483 |
| Functional maturation of kidney organoid tubules: Piezo1-mediated Ca2+ signaling. | Carrisoza-Gaytán, Rolando; Kroll, Katharina T; Hiratsuka, Ken; Gupta, Navin R; Morizane, Ryuji; Lewis, Jennifer A; Satlin, Lisa M | American journal of physiology. Cell physiology | 2023 Feb 06 | 36745528 | |
| Focused ultrasound-mediated brain genome editing. | Leong, Kam; Lao, Yeh-Hsing; Ji, Robin; Zhou, Joyce; Snow, Kathy; Kwon, Nancy; Saville, Ethan; He, Siyu; Chauhan, Shradha; Chi, Chun-Wei; Datta, Malika; Zhang, Hairong; Quek, Chai Hoon; Cai, Sarah; Li, Mingqiang; Gaitan, Yaned; Bechtel, Lawrence; Wu, Shih-Ying; Lutz, Cathleen; Tomer, Raju; Murray, Stephen; Chavez, Alejandro; Konofagou, Elisa | Research square | 2023 Jan 20 | 36712096 | |
| Base editing as a genetic treatment for spinal muscular atrophy. | Alves, Christiano R R; Ha, Leillani L; Yaworski, Rebecca; Lazzarotto, Cicera R; Christie, Kathleen A; Reilly, Aoife; Beauvais, Ariane; Doll, Roman M; de la Cruz, Demitri; Maguire, Casey A; Swoboda, Kathryn J; Tsai, Shengdar Q; Kothary, Rashmi; Kleinstiver, Benjamin P | bioRxiv : the preprint server for biology | 2023 Jan 21 | 36711797 | |
| The Transcriptional Response to Lung-Targeting Lipid Nanoparticles in Vivo. | Radmand, Afsane; Lokugamage, Melissa P; Kim, Hyejin; Dobrowolski, Curtis; Zenhausern, Ryan; Loughrey, David; Huayamares, Sebastian G; Hatit, Marine Z C; Ni, Huanzhen; Del Cid, Ada; Da Silva Sanchez, Alejandro J; Paunovska, Kalina; Schrader Echeverri, Elisa; Shajii, Aram; Peck, Hannah; Santangelo, Philip J; Dahlman, James E | Nano letters | 2023 Feb 08 | 993-1002 | 36701517 |
| Clinical progress in genome-editing technology and in vivo delivery techniques. | Khirallah, Jennifer; Eimbinder, Maximilan; Li, Yamin; Xu, Qiaobing | Trends in genetics : TIG | 2023 Jan 18 | 36669950 | |
| Phenylboronic Acid-Functionalized Polyplexes Tailored to Oral CRISPR Delivery. | Yoshinaga, Naoto; Zhou, Joyce K; Xu, Cong; Quek, Chai Hoon; Zhu, Yuefei; Tang, Ding; Hung, Lin Yung; Najjar, Sarah A; Shiu, Chin Ying Angela; Margolis, Kara Gross; Lao, Yeh-Hsing; Leong, Kam W | Nano letters | 2023 Feb 08 | 757-764 | 36648291 |
| Synthetic Biology Design as a Paradigm Shift toward Manufacturing Affordable Adeno-Associated Virus Gene Therapies. | Collins, Logan Thrasher; Ponnazhagan, Selvarangan; Curiel, David T | ACS synthetic biology | 2023 Jan 20 | 17-26 | 36627108 |
| Accounting for diversity in the design of CRISPR-based therapeutic genome editing. | Saha, Krishanu | Nature genetics | 2023 Jan | 6-7 | 36593304 |
| In Vivo Cellular Magnetic Imaging: Labeled vs. Unlabeled Cells. | Bulte, Jeff W M; Wang, Chao; Shakeri-Zadeh, Ali | Advanced functional materials | 2022 Dec 09 | 36589903 | |
| In vivo editing of the pan-endothelium by immunity evading simian adenoviral vector. | Lorincz, Reka; Alvarez, Aluet Borrego; Walkey, Christopher J; Mendonça, Samir A; Lu, Zhi Hong; Martinez, Alexa E; Ljungberg, Cecilia; Heaney, Jason D; Lagor, William R; Curiel, David T | Biomedicine & pharmacotherapy = Biomedecine & pharmacotherapie | 2023 Feb | 114189 | 36587560 |
| Efficient in vivo neuronal genome editing in the mouse brain using nanocapsules containing CRISPR-Cas9 ribonucleoproteins. | Metzger, Jeanette M; Wang, Yuyuan; Neuman, Samuel S; Snow, Kathy J; Murray, Stephen A; Lutz, Cathleen M; Bondarenko, Viktoriya; Felton, Jesi; Gimse, Kirstan; Xie, Ruosen; Li, Dongdong; Zhao, Yi; Flowers, Matthew T; Simmons, Heather A; Roy, Subhojit; Saha, Krishanu; Levine, Jon E; Emborg, Marina E; Gong, Shaoqin | Biomaterials | 2022 Dec 13 | 36527789 | |
| Reframing the perception of outliers and negative data in translational research. | Emborg, Marina E | Brain research bulletin | 2023 Jan | 203-207 | 36464129 |
| Cyclin G1 induces maladaptive proximal tubule cell dedifferentiation and renal fibrosis through CDK5 activation. | Taguchi, Kensei; Elias, Bertha C; Sugahara, Sho; Sant, Snehal; Freedman, Benjamin S; Waikar, Sushrut S; Pozzi, Ambra; Zent, Roy; Harris, Raymond C; Parikh, Samir M; Brooks, Craig R | The Journal of clinical investigation | 2022 Dec 01 | 36453545 | |
| Substituting racemic ionizable lipids with stereopure ionizable lipids can increase mRNA delivery. | Da Silva Sanchez, Alejandro J; Zhao, Kun; Huayamares, Sebastian G; Hatit, Marine Z C; Lokugamage, Melissa P; Loughrey, David; Dobrowolski, Curtis; Wang, Shuaishuai; Kim, Hyejin; Paunovska, Kalina; Kuzminich, Yanina; Dahlman, James E | Journal of controlled release : official journal of the Controlled Release Society | 2022 Nov 30 | 36423872 | |
| Diverse virus-encoded CRISPR-Cas systems include streamlined genome editors. | Al-Shayeb, Basem; Skopintsev, Petr; Soczek, Katarzyna M; Stahl, Elizabeth C; Li, Zheng; Groover, Evan; Smock, Dylan; Eggers, Amy R; Pausch, Patrick; Cress, Brady F; Huang, Carolyn J; Staskawicz, Brian; Savage, David F; Jacobsen, Steven E; Banfield, Jillia | Cell | 2022 Nov 23 | 36423580 | |
| ABE8e adenine base editor precisely and efficiently corrects a recurrent COL7A1 nonsense mutation. | Sheriff, Adam; Guri, Ina; Zebrowska, Paulina; Llopis-Hernandez, Virginia; Brooks, Imogen R; Tekkela, Stavroula; Subramaniam, Kavita; Gebrezgabher, Ruta; Naso, Gaetano; Petrova, Anastasia; Balon, Katarzyna; Onoufriadis, Alexandros; Kujawa, Dorota; Kotulska, Martyna; Newby, Gregory; Łaczmański, Łukasz; Liu, David R; McGrath, John A; Jacków, Joanna | Scientific reports | 2022 Nov 16 | 36385635 | |
| Production and characterization of virus-free, CRISPR-CAR T cells capable of inducing solid tumor regression. | Mueller, Katherine P; Piscopo, Nicole J; Forsberg, Matthew H; Saraspe, Louise A; Das, Amritava; Russell, Brittany; Smerchansky, Madeline; Cappabianca, Dan; Shi, Lei; Shankar, Keerthana; Sarko, Lauren; Khajanchi, Namita; La Vonne Denne, Nina; Ramamurthy, A | Journal for immunotherapy of cancer | 2022 Sep | 36382633 | |
| LPA disruption with AAV-CRISPR potently lowers plasma apo(a) in transgenic mouse model: A proof-of-concept study. | Doerfler, Alexandria M; Park, So Hyun; Assini, Julia M; Youssef, Amer; Saxena, Lavanya; Yaseen, Adam B; De Giorgi, Marco; Chuecos, Marcel; Hurley, Ayrea E; Li, Ang; Marcovina, Santica M; Bao, Gang; Boffa, Michael B; Koschinsky, Marlys L; Lagor, William R | Molecular therapy. Methods & clinical development | 2022 Dec 08 | 36381302 | |
| Evolution of an adenine base editor into a small, efficient cytosine base editor with low off-target activity. | Neugebauer, Monica E; Hsu, Alvin; Arbab, Mandana; Krasnow, Nicholas A; McElroy, Amber N; Pandey, Smriti; Doman, Jordan L; Huang, Tony P; Raguram, Aditya; Banskota, Samagya; Newby, Gregory A; Tolar, Jakub; Osborn, Mark J; Liu, David R | Nature biotechnology | 2022 Nov 10 | 36357719 | |
| Prime editing for precise and highly versatile genome manipulation. | Chen, Peter J; Liu, David R | Nature reviews. Genetics | 2023 Mar | 161-177 | 36344749 |
| Adult human kidney organoids originate from CD24+ cells and represent an advanced model for adult polycystic kidney disease. | Xu, Yaoxian; Kuppe, Christoph; Perales-Patón, Javier; Hayat, Sikander; Kranz, Jennifer; Abdallah, Ali T; Nagai, James; Li, Zhijian; Peisker, Fabian; Saritas, Turgay; Halder, Maurice; Menzel, Sylvia; Hoeft, Konrad; Kenter, Annegien; Kim, Hyojin; van Roeyen, Claudia R C; Lehrke, Michael; Moellmann, Julia; Speer, Thimoteus; Buhl, Eva M; Hoogenboezem, Remco; Boor, Peter; Jansen, Jitske; Knopp, Cordula; Kurth, Ingo; Smeets, Bart; Bindels, Eric; Reinders, Marlies E J; Baan, Carla; Gribnau, Joost; Hoorn, Ewout J; Steffens, Joachim; Huber, Tobias B; Costa, Ivan; Floege, Jürgen; Schneider, Rebekka K; Saez-Rodriguez, Julio; Freedman, Benjamin S; Kramann, Rafael | Nature genetics | 2022 Oct 27 | 36303074 | |
| Unadjuvanted intranasal spike vaccine elicits protective mucosal immunity against sarbecoviruses. | Mao, Tianyang; Israelow, Benjamin; Peña-Hernández, Mario A; Suberi, Alexandra; Zhou, Liqun; Luyten, Sophia; Reschke, Melanie; Dong, Huiping; Homer, Robert J; Saltzman, W Mark; Iwasaki, Akiko | Science (New York, N.Y.) | 2022 Nov 25 | 36302057 | |
| Nucleic Acid Delivery to the Vascular Endothelium. | Reschke, Melanie; Piotrowski-Daspit, Alexandra S; Pober, Jordan S; Saltzman, W Mark | Molecular pharmaceutics | 2022 Dec 05 | 36251765 | |
| Translating musculoskeletal bioengineering into tissue regeneration therapies. | Khodabukus, Alastair; Guyer, Tyler; Moore, Axel C; Stevens, Molly M; Guldberg, Robert E; Bursac, Nenad | Science translational medicine | 2022 Oct 12 | 36223445 | |
| Monobody adapter for functional antibody display on nanoparticles for adaptable targeted delivery applications. | Albert, C; Bracaglia, L; Koide, A; DiRito, J; Lysyy, T; Harkins, L; Edwards, C; Richfield, O; Grundler, J; Zhou, K; Denbaum, E; Ketavarapu, G; Hattori, T; Perincheri, S; Langford, J; Feizi, A; Haakinson, D; Hosgood, S A; Nicholson, M L; Pober, J S; Saltzman, W M; Koide, S; Tietjen, G T | Nature communications | 2022 Oct 11 | 36220817 | |
| EZ Clear for simple, rapid, and robust mouse whole organ clearing. | Hsu, Chih-Wei; Cerda, Juan; Kirk, Jason M; Turner, Williamson D; Rasmussen, Tara L; Flores Suarez, Carlos P; Dickinson, Mary E; Wythe, Joshua D | eLife | 2022 Oct 11 | 36218247 | |
| Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing. | Gonzalez, Trevor J; Simon, Katherine E; Blondel, Leo O; Fanous, Marco M; Roger, Angela L; Maysonet, Maribel Santiago; Devlin, Garth W; Smith, Timothy J; Oh, Daniel K; Havlik, L Patrick; Castellanos Rivera, Ruth M; Piedrahita, Jorge A; ElMallah, Mai K; Ger | Nature communications | 2022 Oct 10 | 36210364 | |
| In vivo correction of cystic fibrosis mediated by PNA nanoparticles. | Piotrowski-Daspit, Alexandra S; Barone, Christina; Lin, Chun-Yu; Deng, Yanxiang; Wu, Douglas; Binns, Thomas C; Xu, Emily; Ricciardi, Adele S; Putman, Rachael; Garrison, Alannah; Nguyen, Richard; Gupta, Anisha; Fan, Rong; Glazer, Peter M; Saltzman, W Mark; Egan, Marie E | Science advances | 2022 Oct 07 | eabo0522 | 36197984 |
| Direct ITR-to-ITR Nanopore Sequencing of AAV Vector Genomes. | Namkung, Suk; Tran, Ngoc Tam; Manokaran, Sangeetha; He, Ran; Su, Qin; Xie, Jun; Gao, Guangping; Tai, Phillip W L | Human gene therapy | 2022 Nov | 36178359 | |
| Engineering multicellular living systems-A Keystone Symposia report. | Cable, Jennifer; Arlotta, Paola; Parker, Kevin Kit; Hughes, Alex J; Goodwin, Katharine; Mummery, Christine L; Kamm, Roger D; Engle, Sandra J; Tagle, Danilo A; Boj, Sylvia F; Stanton, Alice E; Morishita, Yoshihiro; Kemp, Melissa L; Norfleet, Dennis A; May, | Annals of the New York Academy of Sciences | 2022 Sep 30 | 36177947 | |
| Organoid-on-a-chip model of human ARPKD reveals mechanosensing pathomechanisms for drug discovery. | Hiratsuka, Ken; Miyoshi, Tomoya; Kroll, Katharina T; Gupta, Navin R; Valerius, M Todd; Ferrante, Thomas; Yamashita, Michifumi; Lewis, Jennifer A; Morizane, Ryuji | Science advances | 2022 09 23 | 36129975 | |
| Promoting validation and cross-phylogenetic integration in model organism research. | Cheng, Keith C; Burdine, Rebecca D; Dickinson, Mary E; Ekker, Stephen C; Lin, Alex Y; Lloyd, K C Kent; Lutz, Cathleen M; MacRae, Calum A; Morrison, John H; O'Connor, David H; Postlethwait, John H; Rogers, Crystal D; Sanchez, Susan; Simpson, Julie H; Talbot, William S; Wallace, Douglas C; Weimer, Jill M; Bellen, Hugo J | Disease models & mechanisms | 2022 09 01 | 36125045 | |
| Precision genome editing in the eye. | Suh, Susie; Choi, Elliot H; Raguram, Aditya; Liu, David R; Palczewski, Krzysztof | Proceedings of the National Academy of Sciences of the United States of America | 2022 Sep 27 | 36122230 | |
| Differential microRNA profiles of intramuscular and secreted extracellular vesicles in human tissue-engineered muscle. | Vann, Christopher G; Zhang, Xin; Khodabukus, Alastair; Orenduff, Melissa C; Chen, Yu-Hsiu; Corcoran, David L; Truskey, George A; Bursac, Nenad; Kraus, Virginia B | Frontiers in physiology | 2022 | 937899 | 36091396 |
| 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 R | Nature biotechnology | 2022 Sep 08 | 36076084 | |
| An ELISA-based platform for rapid identification of structure-dependent nucleic acid-protein interactions detects novel DNA triplex interactors. | Economos, Nicholas G; Thapar, Upasna; Balasubramanian, Nanda; Karras, Georgios I; Glazer, Peter M | The Journal of biological chemistry | 2022 Oct | 35988651 | |
| Prostate cancer cells demonstrate unique metabolism and substrate adaptability acutely after androgen deprivation therapy. | Filon, Mikolaj J; Gillette, Amani A; Yang, Bing; Khemees, Tariq A; Skala, Melissa C; Jarrard, David F | The Prostate | 2022 Dec | 35980831 | |
| 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 E | Nature communications | 2022 08 15 | 35970837 | |
| Material Engineering in Gut Microbiome and Human Health. | Yang, Letao; Hung, Lin Y; Zhu, Yuefei; Ding, Suwan; Margolis, Kara G; Leong, Kam W | Research (Washington, D.C.) | 2022 | 9804014 | 35958108 |
| Designing and executing prime editing experiments in mammalian cells. | Doman, Jordan L; Sousa, Alexander A; Randolph, Peyton B; Chen, Peter J; Liu, David R | Nature protocols | 2022 Aug 08 | 35941224 | |
| Multiomics characterization of mouse hepatoblastoma identifies yes-associated protein 1 target genes. | Rodríguez, Tomás C; Kwan, Suet-Yan; Smith, Jordan L; Dadafarin, Sina; Wu, Chern-Horng; Sontheimer, Erik J; Xue, Wen | Hepatology (Baltimore, Md.) | 2022 Aug 06 | 35932276 | |
| 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 R | Nature biomedical engineering | 2022 Jul 28 | 35902773 | |
| 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 J | Molecular pharmaceutics | 2022 08 01 | 35857429 | |
| Non-Invasive imaging of extracellular vesicles: Quo vaditis in vivo? | Arifin, Dian R; Witwer, Kenneth W; Bulte, Jeff W M | Journal of extracellular vesicles | 2022 Jul | 35844061 | |
| 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 S | Methods in molecular biology (Clifton, N.J.) | 2022 | 35838970 | |
| SARS-CoV-2 Host Immunogenetic Biomarkers. | Gededzha, Maemu P; Mampeule, Nakampe; Gandini, Anastasia; Mayne, Elizabeth S | Methods in molecular biology (Clifton, N.J.) | 2022 | 35838957 | |
| Therapeutic in vivo delivery of gene editing agents. | Raguram, Aditya; Banskota, Samagya; Liu, David R | Cell | 2022 Jul 21 | 35798006 | |
| 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 C | Biomedical optics express | 2022 Jun 01 | 35781966 | |
| Nanoparticle single-cell multiomic readouts reveal that cell heterogeneity influences lipid nanoparticle-mediated messenger RNA delivery. | Dobrowolski, Curtis; Paunovska, Kalina; Schrader Echeverri, Elisa; Loughrey, David; Da Silva Sanchez, Alejandro J; Ni, Huanzhen; Hatit, Marine Z C; Lokugamage, Melissa P; Kuzminich, Yanina; Peck, Hannah E; Santangelo, Philip J; Dahlman, James E | Nature nanotechnology | 2022 08 | 35768613 | |
| Kidney organoids: a pioneering model for kidney diseases. | Tekguc, Murat; Gaal, Ronald C VAN; Uzel, Sebastien G M; Gupta, Navin; Riella, Leonardo V; Lewis, Jennifer A; Morizane, Ryuji | Translational research : the journal of laboratory and clinical medicine | 2022 Jun 21 | 35750295 | |
| 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 T | ACS nano | 2022 Jun 24 | 35749339 | |
| The balance between protective and pathogenic immune responses to pneumonia in the neonatal lung is enforced by gut microbiota. | Stevens, Joseph; Steinmeyer, Shelby; Bonfield, Madeline; Peterson, Laura; Wang, Timothy; Gray, Jerilyn; Lewkowich, Ian; Xu, Yan; Du, Yina; Guo, Minzhe; Wynn, James L; Zacharias, William; Salomonis, Nathan; Miller, Lisa; Chougnet, Claire; O'Connor, Dennis Hartigan; Deshmukh, Hitesh | Science translational medicine | 2022 Jun 15 | eabl3981 | 35704600 |
| 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; Ste | Nature biotechnology | 2022 Aug | 35681059 | |
| Synthesis and ex vivo biodistribution of two 68Ga-labeled tetrazine tracers: Comparison of pharmacokinetics. | Lambidis, Elisavet; Lumén, Dave; Koskipahta, Elina; Imlimthan, Surachet; Lopez, Brianda B; Sánchez, Ana Isabel Fraguas; Sarparanta, Mirkka; Cheng, R Holland; Airaksinen, Anu J | Nuclear medicine and biology | 2022 | 35680503 | |
| 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 C | Journal of biomedical optics | 2022 05 | 35643815 | |
| Imaging the fetal nonhuman primate brain with SV2A positron emission tomography (PET). | Rossano, Samantha; Toyonaga, Takuya; Berg, Eric; Lorence, Isabella; Fowles, Krista; Nabulsi, Nabeel; Ropchan, Jim; Li, Songye; Ye, Yunpeng; Felchner, Zachary; Kukis, David; Huang, Yiyun; Benveniste, Helene; Tarantal, Alice F; Groman, Stephanie; Carson, Richard E | European journal of nuclear medicine and molecular imaging | 2022 09 | 35633376 | |
| CRISPR Library Screening in Cultured Cardiomyocytes. | DeLuca, Sophia; Bursac, Nenad | Methods in molecular biology (Clifton, N.J.) | 2022 | 35618895 | |
| 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, Krishanu | GEN biotechnology | 2022 Apr 01 | 35586336 | |
| 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 E | Nature cardiovascular research | 2022 Apr | 35571675 | |
| Structural biology of CRISPR-Cas immunity and genome editing enzymes. | Wang, Joy Y; Pausch, Patrick; Doudna, Jennifer A | Nature reviews. Microbiology | 2022 11 | 35562427 | |
| The clinical progress of mRNA vaccines and immunotherapies. | Barbier, Ann J; Jiang, Allen Yujie; Zhang, Peng; Wooster, Richard; Anderson, Daniel G | Nature biotechnology | 2022 06 | 35534554 | |
| 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 P | Nature genetics | 2022 05 | 35513721 | |
| Identification and Validation of CRISPR/Cas9 Off-Target Activity in Hematopoietic Stem and Progenitor Cells. | Park, So Hyun; Lee, Ciaran M; Bao, Gang | Methods in molecular biology (Clifton, N.J.) | 2022 | 35507169 | |
| 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 C | STAR protocols | 2022 Jun 17 | 35496789 | |
| 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 S | Nature biomedical engineering | 2022 04 | 35478224 | |
| Tetrazine-Ligated CRISPR sgRNAs for Efficient Genome Editing. | Chen, Zexiang; Devi, Gitali; Arif, Amena; Zamore, Phillip D; Sontheimer, Erik J; Watts, Jonathan K | ACS chemical biology | 2022 05 20 | 35446558 | |
| Crystal structure of an RNA/DNA strand exchange junction. | Cofsky, Joshua C; Knott, Gavin J; Gee, Christine L; Doudna, Jennifer A | PloS one | 2022 | 35436289 | |
| CRISPR-Cas9 bends and twists DNA to read its sequence. | Cofsky, Joshua C; Soczek, Katarzyna M; Knott, Gavin J; Nogales, Eva; Doudna, Jennifer A | Nature structural & molecular biology | 2022 Apr | 35422516 | |
| 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, Nenad | Biomaterials | 2022 05 | 35421801 | |
| 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, David | Science translational medicine | 2022 Apr 12 | 35412328 | |
| 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 H | PloS one | 2022 | 35385514 | |
| 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, Krzysztof | Nature communications | 2022 Apr 05 | 35383196 | |
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| 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 R | Nature biotechnology | 2022 Apr 04 | 35379961 | |
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| 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, Deepak | Cell | 2022 03 03 | 35182466 | |
| 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 J | Development (Cambridge, England) | 2022 04 15 | 35178561 | |
| 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, Qiaobing | Proceedings of the National Academy of Sciences of the United States of America | 2022 02 22 | 35173043 | |
| Intestinal Deletion of 3-Hydroxy-3-Methylglutaryl-Coenzyme A Reductase Promotes Expansion of the Resident Stem Cell Compartment. | Doerfler, Alexandria M; Han, Jun; Jarrett, Kelsey E; Tang, Li; Jain, Antrix; Saltzman, Alexander; De Giorgi, Marco; Chuecos, Marcel; Hurley, Ayrea E; Li, Ang; Morand, Pauline; Ayala, Claudia; Goodlett, David R; Malovannaya, Anna; Martin, James F; de Aguiar Vallim, Thomas Q; Shroyer, Noah; Lagor, William R | Arteriosclerosis, thrombosis, and vascular biology | 2022 Apr | 35172604 | |
| 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, Niren | Chemical communications (Cambridge, England) | 2022 Mar 03 | 35170593 | |
| Nonhuman Primates in Translational Research. | Tarantal, Alice F; Noctor, Stephen C; Hartigan-O'Connor, Dennis J | Annual review of animal biosciences | 2022 02 15 | 35167321 | |
| 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 A | Molecular imaging and biology | 2022 04 | 35146614 | |
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| 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, Shaoqin | Advanced materials (Deerfield Beach, Fla.) | 2022 Feb 04 | 35119139 | |
| 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, Wen | Nature communications | 2022 01 21 | 35064134 | |
| Detecting Cardiovascular Protein-Protein Interactions by Proximity Proteomics. | Circulation research | 2022 01 21 | 35050691 | ||
| 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, Martin | Nature cell biology | 2022 01 | 35027731 | |
| 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 R | Cell | 2022 01 20 | 35021064 | |
| 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, Wen | Molecular therapy : the journal of the American Society of Gene Therapy | 2022 Jan 05 | 34998953 | |
| Editorial: "AAV Gene Therapy: Immunology and Immunotherapeutics". | Martinez-Navio, José M; Paulk, Nicole K; Gao, Guangping | Frontiers in immunology | 2021 | 34992614 | |
| rAAV-delivered PTEN therapeutics for prostate cancer. | Ai, Jianzhong; Li, Jia; Su, Qin; Ma, Hong; Wei, Qiang; Li, Hong; Gao, Guangping | Molecular therapy. Nucleic acids | 2022 Mar 08 | 34976432 | |
| CRISPR/Cas9 ribonucleoprotein-mediated genome and epigenome editing in mammalian cells. | Advanced drug delivery reviews | 2022 Feb | 34942274 | ||
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| 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 Travis | Circulation | 2022 01 18 | 34905694 | |
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| 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 R | Nature biotechnology | 2021 Dec 09 | 34887556 | |
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| 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, Niren | Advanced healthcare materials | 2021 Dec 03 | 34861744 | |
| 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 C | The CRISPR journal | 2021 12 | 34847747 | |
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| 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, Wen | Molecular therapy : the journal of the American Society of Gene Therapy | 2021 Oct 23 | 34695545 | |
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| 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 Mark | ACS nano | 2021 10 26 | 34633171 | |
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| 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 W | Materials today (Kidlington, England) | 2021 Jan-Feb | 34421329 | |
| Synthetic Biology Approaches for Engineering Next-Generation Adenoviral Gene Therapies. | Collins, Logan Thrasher; Curiel, David T | ACS nano | 2021 09 28 | 34415739 | |
| Easy-Prime: a machine learning-based prime editor design tool. | Li, Yichao; Chen, Jingjing; Tsai, Shengdar Q; Cheng, Yong | Genome biology | 2021 08 19 | 34412673 | |
| 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, Gordana | Biomaterials | 2021 09 | 34403849 | |
| 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 W | Advanced science (Weinheim, Baden-Wurttemberg, Germany) | 2021 08 | 34386315 | |
| 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 A | Nature structural & molecular biology | 2021 08 | 34381246 | |
| 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 Travis | Cell reports | 2021 Aug 10 | 34380038 | |
| 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 A | Nature methods | 2021 08 | 34341582 | |
| Label-free imaging for quality control of cardiomyocyte differentiation. | Qian, Tongcheng; Heaster, Tiffany M; Houghtaling, Angela R; Sun, Kexin; Samimi, Kayvan; Skala, Melissa C | Nature communications | 2021 07 28 | 34321477 | |
| Vector Strategies to Actualize B Cell-Based Gene Therapies. | Jeske, Amanda M; Boucher, Paul; Curiel, David T; Voss, James E | Journal of immunology (Baltimore, Md. : 1950) | 2021 08 01 | 34321286 | |
| External stimuli-responsive nanoparticles for spatially and temporally controlled delivery of CRISPR-Cas genome editors. | Xie, Ruosen; Wang, Yuyuan; Gong, Shaoqin | Biomaterials science | 2021 Sep 14 | 34286726 | |
| Recent innovations in fluorescence lifetime imaging microscopy for biology and medicine. | Datta, Rupsa; Gillette, Amani; Stefely, Matthew; Skala, Melissa C | Journal of biomedical optics | 2021 07 | 34247457 | |
| 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, Aravind | Journal of virology | 2021 09 09 | 34232726 | |
| 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, Guangping | Gene therapy | 2021 Jul 06 | 34226687 | |
| Mucopolysaccharidoses type I gene therapy. | Hurt, Sarah C; Dickson, Patricia I; Curiel, David T | Journal of inherited metabolic disease | 2021 Jun 29 | 34189746 | |
| 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 R | Nature biotechnology | 2021 Jun 28 | 34183861 | |
| 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, Yilai | Molecular therapy : the journal of the American Society of Gene Therapy | 2021 Jun 24 | 34174443 | |
| Controlling CRISPR with small molecule regulation for somatic cell genome editing. | Khajanchi, Namita; Saha, Krishanu | Molecular therapy : the journal of the American Society of Gene Therapy | 2021 Jun 24 | 34174442 | |
| 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 E | Molecular therapy : the journal of the American Society of Gene Therapy | 2022 01 05 | 34174439 | |
| 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, Shaoqin | Journal of controlled release : official journal of the Controlled Release Society | 2021 Jun 23 | 34174352 | |
| Magnetic Forces Enable Control of Biological Processes In Vivo. | Bao, Gang | Journal of applied mechanics | 2021 Mar 01 | 34168385 | |
| 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 R | Molecular therapy. Methods & clinical development | 2021 Jun 11 | 34141821 | |
| 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, Kan | Aging cell | 2021 07 | 34086398 | |
| 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 R | Nature | 2021 Jul | 34079130 | |
| Escaping the endosome: assessing cellular trafficking mechanisms of non-viral vehicles. | Xu, Emily; Saltzman, W Mark; Piotrowski-Daspit, Alexandra S | Journal of controlled release : official journal of the Controlled Release Society | 2021 07 10 | 34077782 | |
| 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 A | Cell reports | 2021 Jun 01 | 34077734 | |
| 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 Mohan | Blood | 2021 07 29 | 34075401 | |
| 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, Jun | Genes | 2021 05 25 | 34070435 | |
| 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 C | Biosensors & bioelectronics | 2021 Oct 01 | 34062334 | |
| Quantification of Cellular Densities and Antigenic Properties using Magnetic Levitation. | Thompson, Lauren; Pinckney, Brandy; Lu, Shulin; Gregory, Mark; Tigges, John; Ghiran, Ionita | Journal of visualized experiments : JoVE | 2021 05 17 | 34057455 | |
| Vectored Immunotherapeutics for Infectious Diseases: Can rAAVs Be The Game Changers for Fighting Transmissible Pathogens? | Zhan, Wei; Muhuri, Manish; Tai, Phillip W L; Gao, Guangping | Frontiers in immunology | 2021 | 34046041 | |
| 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, Guoping | Cell | 2021 Jun 10 | 34043941 | |
| 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, Guangping | Frontiers in immunology | 2021 | 33995418 | |
| 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-Mei | Clinical and translational science | 2021 09 | 33982436 | |
| 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, Nenad | Communications biology | 2021 05 05 | 33953320 | |
| 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; Hins | Cell reports | 2021 May 04 | 33951429 | |
| Integrating Biomaterials and Genome Editing Approaches to Advance Biomedical Science. | Abdeen, Amr A; Cosgrove, Brian D; Gersbach, Charles A; Saha, Krishanu | Annual review of biomedical engineering | 2021 07 13 | 33909475 | |
| VectorMOD: Method for Bottom-Up Proteomic Characterization of rAAV Capsid Post-Translational Modifications and Vector Impurities. | Rumachik, Neil G; Malaker, Stacy A; Paulk, Nicole K | Frontiers in immunology | 2021 | 33868302 | |
| 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, Wen | Nature communications | 2021 04 09 | 33837189 | |
| 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 Consortium | Nature | 2021 Apr | 33828315 | |
| Examining the evidence for extracellular RNA function in mammals. | Gruner, Hannah N; McManus, Michael T | Nature reviews. Genetics | 2021 07 | 33824487 | |
| 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 Mark | Biomaterials | 2021 May | 33813260 | |
| 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, Guangping | Oncogene | 2021 Apr | 33782545 | |
| 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 B | Molecular therapy. Methods & clinical development | 2021 Jun 11 | 33768133 | |
| 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 E | Bioengineering & translational medicine | 2020 Sep | 33758781 | |
| 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 C | Science translational medicine | 2021 04 21 | 33723017 | |
| 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 M | Genome biology | 2021 03 16 | 33722289 | |
| Multicellular Systems to Translate Somatic Cell Genome Editors to Humans. | Hernandez-Gordillo, Victor; Casolaro, Thomas Caleb; Ebrahimkhani, Mo R; Kiani, Samira | Current opinion in biomedical engineering | 2020 Dec | 33718690 | |
| Tissue-Engineered Skeletal Muscle Models to Study Muscle Function, Plasticity, and Disease. | Khodabukus, Alastair | Frontiers in physiology | 2021 | 33716768 | |
| AAV-Mediated Gene Therapy for Glycosphingolipid Biosynthesis Deficiencies. | Yang, Huiya; Brown Jr, Robert H; Wang, Dan; Strauss, Kevin A; Gao, Guangping | Trends in molecular medicine | 2021 06 | 33714697 | |
| 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, Nenad | Acta biomaterialia | 2021 05 | 33705988 | |
| 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 A | Nucleic acids research | 2021 04 06 | 33693715 | |
| Therapeutic RNA Delivery for COVID and Other Diseases. | Dobrowolski, Curtis; Paunovska, Kalina; Hatit, Marine Z C; Lokugamage, Melissa P; Dahlman, James E | Advanced healthcare materials | 2021 Mar 04 | 33661555 | |
| 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, Qiaobing | Proceedings of the National Academy of Sciences of the United States of America | 2021 Mar 09 | 33649229 | |
| 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 E | eLife | 2021 02 23 | 33620031 | |
| 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, Aravind | Molecular therapy. Nucleic acids | 2021 Mar 05 | 33614232 | |
| 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 G | Cell | 2021 02 18 | 33606977 | |
| 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, Luca | Nature communications | 2021 02 15 | 33589617 | |
| 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, Yoshikazu | The Journal of biological chemistry | 2021 Feb 08 | 33571526 | |
| Viral vector platforms within the gene therapy landscape. | Bulcha, Jote T; Wang, Yi; Ma, Hong; Tai, Phillip W L; Gao, Guangping | Signal transduction and targeted therapy | 2021 Feb 08 | 33558455 | |
| Gene-based therapies for neurodegenerative diseases. | Sun, Jichao; Roy, Subhojit | Nature neuroscience | 2021 03 | 33526943 | |
| 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, Nenad | Science advances | 2021 Jan | 33523949 | |
| 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 H | Acta biomaterialia | 2021 Jan 21 | 33484911 | |
| 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, Pan | The Journal of prosthetic dentistry | 2021 Jan 16 | 33468316 | |
| Precision genome editing using cytosine and adenine base editors in mammalian cells. | Huang, Tony P; Newby, Gregory A; Liu, David R | Nature protocols | 2021 Feb | 33462442 | |
| CRISPR/Cas9 gene editing for curing sickle cell disease. | Park, So Hyun; Bao, Gang | Transfusion and apheresis science : official journal of the World Apheresis Association : official journal of the European Society for Haemapheresis | 2021 Jan 10 | 33455878 | |
| 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 R | Nature | 2021 Jan | 33408413 | |
| 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, Guangping | The Journal of clinical investigation | 2021 Jan 04 | 33393506 | |
| 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, Wen | Hepatology (Baltimore, Md.) | 2020 Dec 17 | 33336367 | |
| 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 R | Cell systems | 2021 Jan 20 | 33290741 | |
| 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, Gang | Nature 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 A | Cell reports. | 2020 Dec 01; | 33264623 | |
| Phage-assisted continuous and non-continuous evolution. | Miller, Shannon M; Wang, Tina; Liu, David R | Nature protocols. | 2020 Dec; | 33199872 | |
| The once and future gene therapy. | Bulaklak, Karen; Gersbach, Charles A | Nature 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 H | Magnetic 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, Fan | Advanced biosystems | 2020 08 | 33179869 | |
| 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, Olga | Molecular 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 A | Molecular therapy. Methods & clinical development. | 2020 Dec 11; | 33145368 | |
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