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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.
Affiliation
  • Saha K; Department of Biomedical Engineering, University of Wisconsin-Madison, Madison, WI, USA. ksaha@wisc.edu.
  • Sontheimer EJ; Department of Medical History & Bioethics, University of Wisconsin-Madison, Madison, WI, USA. ksaha@wisc.edu.
  • Brooks PJ; Wisconsin Institute for Discovery, University of Wisconsin-Madison, Madison, WI, USA. ksaha@wisc.edu.
  • Dwinell MR; McPherson Eye Research Institute, University of Wisconsin-Madison, Madison, WI, USA. ksaha@wisc.edu.
  • Gersbach CA; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA. erik.sontheimer@umassmed.edu.
  • Liu DR; Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health, Bethesda, MD, USA.
  • Murray SA; Department of Physiology, Medical College of Wisconsin, Milwaukee, WI, USA.
  • Tsai SQ; Department of Biomedical Engineering, Duke University, Durham, NC, USA.
  • Wilson RC; Merkin Institute of Transformative Technologies, Broad Institute of MIT and Harvard, Cambridge, MA, USA.
  • Anderson DG; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA.
  • Asokan A; Howard Hughes Medical Institute, Cambridge, MA, USA.
  • Banfield JF; The Jackson Laboratory, Bar Harbor, ME, USA.
  • Bankiewicz KS; Department of Hematology, St Jude Children's Research Hospital, Memphis, TN, USA.
  • Bao G; Innovative Genomics Institute, University of California, Berkeley, Berkeley, CA, USA.
  • Bulte JWM; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA, USA.
  • Bursac N; Institute for Medical Engineering and Science, Massachusetts Institute of Technology, Cambridge, MA, USA.
  • Campbell JM; David H. Koch Institute for Integrative Cancer Research at the Massachusetts Institute of Technology, Cambridge, MA, USA.
  • Carlson DF; Department of Biomedical Engineering, Duke University, Durham, NC, USA.
  • Chaikof EL; Department of Surgery, Duke University School of Medicine, Durham, NC, USA.
  • Chen ZY; Innovative Genomics Institute, University of California, Berkeley, Berkeley, CA, USA.
  • Cheng RH; Department of Earth and Planetary Sciences, University of California, Berkeley, Berkeley, CA, USA.
  • Clark KJ; Department of Neurological Surgery, Ohio State University, Columbus, OH, USA.
  • Curiel DT; Department of Bioengineering, Rice University, Houston, TX, USA.
  • Dahlman JE; Russell H. Morgan Department of Radiology and Radiological Science, Division of MR Research, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
  • Deverman BE; Cellular Imaging Section and Vascular Biology Program, Institute for Cell Engineering, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
  • Dickinson ME; Department of Biomedical Engineering, Duke University, Durham, NC, USA.
  • Doudna JA; Recombinetics, Inc, Eagan, MN, USA.
  • Ekker SC; Recombinetics, Inc, Eagan, MN, USA.
  • Emborg ME; Department of Surgery, Beth Israel Deaconess Medical Center, Boston, MA, USA.
  • Feng G; Department of Otolaryngology-Head and Neck Surgery, Harvard Medical School, Boston, MA, USA.
  • Freedman BS; Program in Neuroscience, Harvard Medical School, Boston, MA, USA.
  • Gamm DM; Eaton-Peabody Laboratory, Massachusetts Eye and Ear Infirmary, Boston, MA, USA.
  • Gao G; Department of Molecular and Cellular Biology, University of California, Davis, Davis, CA, USA.
  • Ghiran IC; Department of Biochemistry and Molecular Biology, Mayo Clinic Rochester, Rochester, MN, USA.
  • Glazer PM; Department of Radiation Oncology, Washington University in St Louis, St Louis, MO, USA.
  • Gong S; Wallace H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology, Atlanta, GA, USA.
  • Heaney JD; Stanley Center for Psychiatric Research, Broad Institute, Cambridge, MA, USA.
  • Hennebold JD; Department of Molecular Physiology and Biophysics, Baylor College of Medicine, Houston, TX, USA.
  • Hinson JT; Innovative Genomics Institute, University of California, Berkeley, Berkeley, CA, USA.
  • Khvorova A; Department of Chemistry, University of California, Berkeley, Berkeley, CA, USA.
  • Kiani S; Department of Molecular and Cell Biology, University of California, Berkeley, Berkeley, CA, USA.
  • Lagor WR; California Institute for Quantitative Biosciences (QB3), University of California, Berkeley, Berkeley, CA, USA.
  • Lam KS; Howard Hughes Medical Institute, University of California, Berkeley, Berkeley, CA, USA.
  • Leong KW; Molecular Biophysics and Integrated Bioimaging Division, Lawrence Berkeley National Laboratory, Berkeley, CA, USA.
  • Levine JE; Gladstone Institute of Data Science and Biotechnology, Gladstone Institutes, San Francisco, CA, USA.
  • Lewis JA; Department of Biochemistry and Molecular Biology, Mayo Clinic Rochester, Rochester, MN, USA.
  • Lutz CM; Department of Medical Physics, University of Wisconsin-Madison, Madison, WI, USA.
  • Ly DH; Wisconsin National Primate Research Center, University of Wisconsin-Madison, Madison, WI, USA.
  • Maragh S; McGovern Institute for Brain Research, Massachusetts Institute of Technology, Cambridge, MA, USA.
  • McCray PB; Division of Nephrology, University of Washington, Seattle, WA, USA.
Nature ; 592(7853): 195-204, 2021 04.
Article in En | MEDLINE | ID: mdl-33828315
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.
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Full text: 1 Database: MEDLINE Main subject: Genome, Human / Cells / Gene Editing / National Institutes of Health (U.S.) Limits: Animals / Humans Country/Region as subject: America do norte Language: En Journal: Nature Year: 2021 Type: Article Affiliation country: United States
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Full text: 1 Database: MEDLINE Main subject: Genome, Human / Cells / Gene Editing / National Institutes of Health (U.S.) Limits: Animals / Humans Country/Region as subject: America do norte Language: En Journal: Nature Year: 2021 Type: Article Affiliation country: United States