The NIH Somatic Cell Genome Editing program

Krishanu Saha (), Erik J. Sontheimer (), P. J. Brooks, Melinda R. Dwinell, Charles A. Gersbach, David R. Liu, Stephen A. Murray, Shengdar Q. Tsai, Ross C. Wilson, Daniel G. Anderson, Aravind Asokan, Jillian F. Banfield, Krystof S. Bankiewicz, Gang Bao, Jeff W. M. Bulte, Nenad Bursac, Jarryd M. Campbell, Daniel F. Carlson, Elliot L. Chaikof, Zheng-Yi Chen, R. Holland Cheng, Karl J. Clark, David T. Curiel, James E. Dahlman, Benjamin E. Deverman, Mary E. Dickinson, Jennifer A. Doudna, Stephen C. Ekker, Marina E. Emborg, Guoping Feng, Benjamin S. Freedman, David M. Gamm, Guangping Gao, Ionita C. Ghiran, Peter M. Glazer, Shaoqin Gong, Jason D. Heaney, Jon D. Hennebold, John T. Hinson, Anastasia Khvorova, Samira Kiani, William R. Lagor, Kit S. Lam, Kam W. Leong, Jon E. Levine, Jennifer A. Lewis, Cathleen M. Lutz, Danith H. Ly, Samantha Maragh, Paul B. McCray, Todd C. McDevitt, Oleg Mirochnitchenko, Ryuji Morizane, Niren Murthy, Randall S. Prather, John A. Ronald, Subhojit Roy, Sushmita Roy, Venkata Sabbisetti, W. Mark Saltzman, Philip J. Santangelo, David J. Segal, Mary Shimoyama, Melissa C. Skala, Alice F. Tarantal, John C. Tilton, George A. Truskey, Moriel Vandsburger, Jonathan K. Watts, Kevin D. Wells, Scot A. Wolfe, Qiaobing Xu, Wen Xue, Guohua Yi and Jiangbing Zhou
Additional contact information
Krishanu Saha: University of Wisconsin-Madison
Erik J. Sontheimer: University of Massachusetts Medical School
P. J. Brooks: National Institutes of Health
Melinda R. Dwinell: Medical College of Wisconsin
Charles A. Gersbach: Duke University
David R. Liu: Broad Institute of MIT and Harvard
Stephen A. Murray: The Jackson Laboratory
Shengdar Q. Tsai: St Jude Children’s Research Hospital
Ross C. Wilson: University of California, Berkeley
Daniel G. Anderson: Massachusetts Institute of Technology
Aravind Asokan: Duke University
Jillian F. Banfield: University of California, Berkeley
Krystof S. Bankiewicz: Ohio State University
Gang Bao: Rice University
Jeff W. M. Bulte: Johns Hopkins University School of Medicine
Nenad Bursac: Duke University
Jarryd M. Campbell: Recombinetics, Inc
Daniel F. Carlson: Recombinetics, Inc
Elliot L. Chaikof: Beth Israel Deaconess Medical Center
Zheng-Yi Chen: Harvard Medical School
R. Holland Cheng: University of California, Davis
Karl J. Clark: Mayo Clinic Rochester
David T. Curiel: Washington University in St Louis
James E. Dahlman: Georgia Institute of Technology
Benjamin E. Deverman: Stanley Center for Psychiatric Research, Broad Institute
Mary E. Dickinson: Baylor College of Medicine
Jennifer A. Doudna: University of California, Berkeley
Stephen C. Ekker: Mayo Clinic Rochester
Marina E. Emborg: University of Wisconsin-Madison
Guoping Feng: Massachusetts Institute of Technology
Benjamin S. Freedman: University of Washington
David M. Gamm: University of Wisconsin-Madison
Guangping Gao: University of Massachusetts Medical School
Ionita C. Ghiran: Beth Israel Deaconess Medical Center
Peter M. Glazer: Yale University
Shaoqin Gong: University of Wisconsin-Madison
Jason D. Heaney: Baylor College of Medicine
Jon D. Hennebold: Oregon National Primate Research Center, Oregon Health and Science University
John T. Hinson: University of Connecticut School of Medicine
Anastasia Khvorova: University of Massachusetts Medical School
Samira Kiani: University of Pittsburgh School of Medicine
William R. Lagor: Baylor College of Medicine
Kit S. Lam: University of California, Davis
Kam W. Leong: Columbia University
Jon E. Levine: University of Wisconsin-Madison
Jennifer A. Lewis: Harvard University
Cathleen M. Lutz: The Jackson Laboratory
Danith H. Ly: Carnegie-Mellon University
Samantha Maragh: National Institute of Standards and Technology
Paul B. McCray: University of Iowa
Todd C. McDevitt: Gladstone Institutes
Oleg Mirochnitchenko: Division of Program Coordination, Planning, and Strategic Initiatives, Office of the Director, National Institutes of Health
Ryuji Morizane: Harvard Medical School
Niren Murthy: University of California, Berkeley
Randall S. Prather: University of Missouri
John A. Ronald: The University of Western Ontario, London
Subhojit Roy: University of California, San Diego
Sushmita Roy: University of Wisconsin-Madison
Venkata Sabbisetti: Brigham and Women’s Hospital
W. Mark Saltzman: Yale University
Philip J. Santangelo: Georgia Institute of Technology
David J. Segal: University of California, Davis
Mary Shimoyama: Marquette University and Medical College of Wisconsin
Melissa C. Skala: University of Wisconsin-Madison
Alice F. Tarantal: University of California, Davis
John C. Tilton: Case Western Reserve University
George A. Truskey: Duke University
Moriel Vandsburger: University of California, Berkeley
Jonathan K. Watts: University of Massachusetts Medical School
Kevin D. Wells: University of Missouri
Scot A. Wolfe: University of Massachusetts Medical School, Worcester
Qiaobing Xu: Tufts University
Wen Xue: University of Massachusetts Medical School
Guohua Yi: University of Texas Health Sciences Center at Tyler
Jiangbing Zhou: Yale University

Nature, 2021, vol. 592, issue 7853, 195-204

Abstract: 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’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.

Date: 2021
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DOI: 10.1038/s41586-021-03191-1

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