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The NIH Somatic Cell Genome Editing program

Author

Listed:
  • Krishanu Saha

    (University of Wisconsin-Madison
    University of Wisconsin-Madison
    University of Wisconsin-Madison
    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
    Harvard University
    Howard Hughes Medical Institute)

  • 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
    Massachusetts Institute of Technology
    David H. Koch Institute for Integrative Cancer Research at the Massachusetts Institute of Technology)

  • Aravind Asokan

    (Duke University
    Duke University School of Medicine)

  • Jillian F. Banfield

    (University of California, Berkeley
    University of California, Berkeley)

  • Krystof S. Bankiewicz

    (Ohio State University)

  • Gang Bao

    (Rice University)

  • Jeff W. M. Bulte

    (Johns Hopkins University School of Medicine
    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
    Harvard Medical School
    Massachusetts Eye and Ear Infirmary)

  • 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
    University of California, Berkeley
    University of California, Berkeley
    University of California, Berkeley)

  • Stephen C. Ekker

    (Mayo Clinic Rochester)

  • Marina E. Emborg

    (University of Wisconsin-Madison
    University of Wisconsin-Madison)

  • Guoping Feng

    (Massachusetts Institute of Technology)

  • Benjamin S. Freedman

    (University of Washington
    University of Washington
    University of Washington
    University of Washington)

  • David M. Gamm

    (University of Wisconsin-Madison
    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
    University of Wisconsin-Madison
    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
    University of California, San Francisco)

  • 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
    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
    University of Wisconsin-Madison
    University of Wisconsin-Madison
    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
    University of Wisconsin-Madison
    Morgridge Institute for Research)

  • Alice F. Tarantal

    (University of California, Davis
    University of California, Davis
    University of California, Davis
    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)

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.

Suggested Citation

  • 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 Asoka, 2021. "The NIH Somatic Cell Genome Editing program," Nature, Nature, vol. 592(7853), pages 195-204, April.
    • Handle: RePEc:nat:nature:v:592:y:2021:i:7853:d:10.1038_s41586-021-03191-1
    DOI: 10.1038/s41586-021-03191-1
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    Cited by:

    1. Katarina Kulhankova & Soumba Traore & Xue Cheng & Hadrien Benk-Fortin & Stéphanie Hallée & Mario Harvey & Joannie Roberge & Frédéric Couture & Sajeev Kohli & Thomas J. Gross & David K. Meyerholz & Gar, 2023. "Shuttle peptide delivers base editor RNPs to rhesus monkey airway epithelial cells in vivo," Nature Communications, Nature, vol. 14(1), pages 1-16, December.
    2. Yuting Chen & Eriona Hysolli & Anlu Chen & Stephen Casper & Songlei Liu & Kevin Yang & Chenli Liu & George Church, 2022. "Multiplex base editing to convert TAG into TAA codons in the human genome," Nature Communications, Nature, vol. 13(1), pages 1-13, December.
    3. Castillejo-Cuberos, A. & Cardemil, J.M. & Escobar, R., 2023. "Techno-economic assessment of photovoltaic plants considering high temporal resolution and non-linear dynamics of battery storage," Applied Energy, Elsevier, vol. 334(C).
    4. Simone Zamin & Leandro José Nichetti & José Antonio Cescon & Vinícius Abílio Martins & Nilton César Lima, 2021. "Lean Practices in the Process of New Product Development: Case of Study in a Food Industry Cooperative in Brazil," Journal of Agricultural Studies, Macrothink Institute, vol. 9(4), pages 41-61, December.
    5. Martin Peterka & Nina Akrap & Songyuan Li & Sandra Wimberger & Pei-Pei Hsieh & Dmitrii Degtev & Burcu Bestas & Jack Barr & Stijn Plassche & Patricia Mendoza-Garcia & Saša Šviković & Grzegorz Sienski &, 2022. "Harnessing DSB repair to promote efficient homology-dependent and -independent prime editing," Nature Communications, Nature, vol. 13(1), pages 1-9, December.
    6. Ioan ADASCALITEI, 2021. "Mobile Security Risks Overview," Informatica Economica, Academy of Economic Studies - Bucharest, Romania, vol. 25(4), pages 53-62.
    7. Zhong, Howard & Hamilton, Mark, 2023. "Exploring gender and race biases in the NFT market," Finance Research Letters, Elsevier, vol. 53(C).

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