@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 = "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",
}