The NIH Somatic Cell Genome Editing program

• Published in: Nature (London) Vol. 592; no. 7853; pp. 195 - 204
• Main Authors: 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
• Format: Journal Article
• Language: English
• Published: London Nature Publishing Group UK 08.04.2021; Nature Publishing Group
• Subjects: 13/100; 13/107; 42/100; 42/44; 45/41; 59/57; 59/78; 631/61/201/2110; 64/60; 692/308/2056; Animal models; Animals; BASIC BIOLOGICAL SCIENCES; Biomedical materials; Cells - metabolism; Consortia; CRISPR; Deoxyribonucleic acid; DNA; Editing; Enzymes; Gene Editing - methods; Gene therapy; Genetic Therapy; Genome editing; Genome, Human - genetics; Genomes; Goals; Humanities and Social Sciences; Humans; In vivo methods and tests; Mitochondrial DNA; multidisciplinary; Mutation; National Institutes of Health (U.S.) - organization & administration; Patients; Perspective; Science; Science (multidisciplinary); Sickle cell disease; Somatic cells; Toolkits; United States; United States; United States > US
• ISSN: 0028-0836; 1476-4687; 1476-4687
• DOI: 10.1038/s41586-021-03191-1

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. This Perspective discusses how the Somatic Cell Genome Editing Consortium aims to accelerate the implementation of safe and effective genome-editing therapies in the clinic.