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, 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

doi:10.1038/s41586-021-03191-1

Title: The NIH Somatic Cell Genome Editing program

Journal Article · Wed Apr 07 00:00:00 EDT 2021 · Nature (London)

DOI:https://doi.org/10.1038/s41586-021-03191-1· OSTI ID:1815941

^[1];

^[2]; Brooks, P. J. ^[3]; Dwinell, Melinda R. ^[4];

^[5];

^[6]; Murray, Stephen A. ^[7];

^[8];

^[9];

^[10];

;

; Bankiewicz, Krystof S.; Bao, Gang; Bulte, Jeff W. M.; Bursac, Nenad; Campbell, Jarryd M.;

;

Univ. of Wisconsin, Madison, WI (United States). Dept. of Biomedical Engineering; Univ. of Wisconsin, Madison, WI (United States). Dept. of Medical History & Bioethics; Univ. of Wisconsin, Madison, WI (United States). Wisconsin Inst. for Discovery; Univ. of Wisconsin, Madison, WI (United States). McPherson Eye Research Inst.
Univ. of Massachusetts Medical School, Worcester, MA (United States). RNA Therapeutics Inst.
National Institutes of Health (NIH), Bethesda, MD (United States). National Center for Advancing Translational Sciences (NCATS). Office of Rare Diseases Research
Medical College of Wisconsin, Milwaukee, WI (United States). Dept. of Physiology
Duke University, Durham, NC (United States). Dept. of Biomedical Engineering
Broad Inst. of MIT and Harvard, Cambridge, MA (United States). Merkin Inst. of Transformative Technologies; Harvard Univ., Cambridge, MA (United States). Dept. of Chemistry and Chemical Biology; Howard Hughes Medical Inst., Cambridge, MA (United States)
The Jackson Lab., Bar Harbor, ME (United States).
St. Jude Children’s Research Hospital, Memphis, TN (United States). Dept. of Hematology
Univ. of California, Berkeley, CA (United States). Innovative Genomics Inst.
Massachusetts Inst. of Technology (MIT), Cambridge, MA (United States). Dept. of of Chemical Engineering. Inst. for Medical Engineering and Science. David H. Koch Inst. for Integrative Cancer Research

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’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.

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Research Organization:: Lawrence Berkeley National Laboratory (LBNL), Berkeley, CA (United States)

Sponsoring Organization:: USDOE Office of Science (SC)

Contributing Organization:: The SCGE Consortium

Grant/Contract Number:: AC02-05CH11231

OSTI ID:: 1815941

Journal Information:: Nature (London), Vol. 592, Issue 7853; ISSN 0028-0836

Publisher:: Nature Publishing GroupCopyright Statement

Country of Publication:: United States

Language:: English

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