@article{a19383e1959b4395870315f5ab48eda2,
title = "The NIH Somatic Cell Genome Editing program",
abstract = "The move from reading to writing the human genome offers new opportunities to improve human health. The United States National Institutes of Health (NIH) Somatic Cell Genome Editing (SCGE) Consortium aims to accelerate the development of safer and more-effective methods to edit the genomes of disease-relevant somatic cells in patients, even in tissues that are difficult to reach. Here we discuss the consortium{\textquoteright}s plans to develop and benchmark approaches to induce and measure genome modifications, and to define downstream functional consequences of genome editing within human cells. Central to this effort is a rigorous and innovative approach that requires validation of the technology through third-party testing in small and large animals. New genome editors, delivery technologies and methods for tracking edited cells in vivo, as well as newly developed animal models and human biological systems, will be assembled—along with validated datasets—into an SCGE Toolkit, which will be disseminated widely to the biomedical research community. We visualize this toolkit—and the knowledge generated by its applications—as a means to accelerate the clinical development of new therapies for a wide range of conditions.",
author = "{The SCGE Consortium} and Krishanu Saha and Sontheimer, {Erik J.} and Brooks, {P. J.} and Dwinell, {Melinda R.} and Gersbach, {Charles A.} and Liu, {David R.} and Murray, {Stephen A.} and Tsai, {Shengdar Q.} and Wilson, {Ross C.} and Anderson, {Daniel G.} and Aravind Asokan and Banfield, {Jillian F.} and Bankiewicz, {Krystof S.} and Gang Bao and Bulte, {Jeff W.M.} and Nenad Bursac and Campbell, {Jarryd M.} and Carlson, {Daniel F.} and Chaikof, {Elliot L.} and Chen, {Zheng Yi} and Cheng, {R. Holland} and Clark, {Karl J.} and Curiel, {David T.} and Dahlman, {James E.} and Deverman, {Benjamin E.} and Dickinson, {Mary E.} and Doudna, {Jennifer A.} and Ekker, {Stephen C.} and Emborg, {Marina E.} and Guoping Feng and Freedman, {Benjamin S.} and Gamm, {David M.} and Guangping Gao and Ghiran, {Ionita C.} and Glazer, {Peter M.} and Shaoqin Gong and Heaney, {Jason D.} and Hennebold, {Jon D.} and Hinson, {John T.} and Anastasia Khvorova and Samira Kiani and Lagor, {William R.} and Lam, {Kit S.} and Leong, {Kam W.} and Levine, {Jon E.} and Lewis, {Jennifer A.} and Lutz, {Cathleen M.} and Ly, {Danith H.} and Samantha Maragh and McCray, {Paul B.}",
note = "Funding Information: Competing interests K.S. receives sponsored research support from Spotlight Therapeutics. E.J.S. is a co-founder and advisor of Intellia Therapeutics. C.A.G. is a co-founder of Tune Therapeutics, Element Genomics and Locus Biosciences, and an advisor to Sarepta Therapeutics, Levo Therapeutics and Iveric Bio. D.R.L. is a co-founder or founder and scientific advisor of Editas Medicine, Pairwise Plants, Beam Therapeutics and Prime Medicine. S.Q.T. is a member of the scientific advisory board of Kromatid, Inc. D.G.A. is a co-founder of CRISPR Therapeutics, Sigilon, Verseau, VasoRx and Orna, and a consultant for Translate Bio and Obsidian Therapeutics. A.A. is a co-founder of StrideBio and TorqueBio, and an advisor to AstraZeneca, Sarepta Therapeutics, Gemini Therapeutics and BridgeBio. J.F.B. is a co-founder of Metagenomi. J.W.M.B. receives research support from NovaDip Biosciences, Philips Healthcare and Weinberg Medical Physics. D.F.C. and J.C. are employees and shareholders in Recombinetics, Inc. J.E.D. is a co-founder of Guide Therapeutics. K.J.C. is a co-founder of Recombinetics, Inc. and LifEngine Technologies. B.E.D. is on the scientific advisory board of Tevard Biosciences. J.A.D. is a co-founder of Caribou Biosciences, Editas Medicine, Scribe Therapeutics, Intellia Therapeutics and Mammoth Biosciences; a scientific advisory board member of Caribou Biosciences, Intellia Therapeutics, eFFECTOR Therapeutics, Scribe Therapeutics, Mammoth Biosciences, Synthego, Algen Biotechnologies, Felix Biosciences and Inari; a Director at Johnson & Johnson; and has research projects sponsored by Biogen, Pfizer, AppleTree Partners and Roche. S.C.E. is co-founder and chief executive officer of LifEngine Technologies, co-founder of LEAH Labs and chief scientific officer of Mettaforge Therapeutics. D.M.G. is a co-founder and chief scientific advisor for Opsis Therapeutics and holds equity in this company, and also receives sponsored research support from Ascidian Therapeutics. G.G. is a scientific co-founder of Voyager Therapeutics, Adrenas Therapeutics and Aspa Therapeutics. P.M.G. is a co-founder of Cybrexa Therapeutics and Delmab Bio and an advisor to pHLIP, Inc. P.B.M. is on the scientific advisory board and receives support for sponsored research from Spirovant Sciences, Inc. S.K. is the chief scientific officer and co-founder of SafeGen Therapeutics. W.M.S. is a co-founder of and receives research support from Stradefy Biosciences. J.K.W. is a consultant for Flagship Pioneering. Publisher Copyright: {\textcopyright} 2021, The Author(s).",
year = "2021",
month = apr,
day = "8",
doi = "10.1038/s41586-021-03191-1",
language = "English",
volume = "592",
pages = "195--204",
journal = "Nature",
issn = "0028-0836",
number = "7853",
}