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In vivo gene editing in dystrophic mouse muscle and muscle stem cells

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Science  22 Jan 2016:
Vol. 351, Issue 6271, pp. 407-411
DOI: 10.1126/science.aad5177

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vol. 351 no. 6271 407-411

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History: 
  • Received for publication September 23, 2015
  • Accepted for publication December 8, 2015

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  1. Mohammadsharif Tabebordbar1,2,*,
  2. Kexian Zhu1,3,*,
  3. Jason K. W. Cheng1,
  4. Wei Leong Chew2,4,
  5. Jeffrey J. Widrick5,
  6. Winston X. Yan6,7,
  7. Claire Maesner1,
  8. Elizabeth Y. Wu1,,
  9. Ru Xiao8,
  10. F. Ann Ran6,7,
  11. Le Cong6,7,
  12. Feng Zhang6,7,
  13. Luk H. Vandenberghe8,
  14. George M. Church4,
  15. Amy J. Wagers1,
  1. 1Department of Stem Cell and Regenerative Biology, Harvard University, and Harvard Stem Cell Institute, Cambridge, MA 02138, USA.
  2. 2Biological and Biomedical Sciences Program, Harvard Medical School, Boston, MA 02115, USA.
  3. 3Department of Molecular and Cellular Biology, Harvard University, Cambridge, MA 02138, USA.
  4. 4Department of Genetics, Harvard Medical School, Boston, MA 02115, USA.
  5. 5Division of Genetics and Program in Genomics, Boston Children’s Hospital, Harvard Medical School, Boston, MA 02115, USA.
  6. 6Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA.
  7. 7McGovern Institute for Brain Research, Department of Brain and Cognitive Science, and Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.
  8. 8Grousbeck Gene Therapy Center, Schepens Eye Research Institute, and Massachusetts Eye and Ear Infirmary, 20 Staniford Street, Boston, MA 02114, USA.
  1. Corresponding author. E-mail: amy_wagers{at}harvard.edu
  1. * These authors contributed equally to this work.

  • Present address: RaNA Therapeutics, 200 Sidney Street, Suite 310, Cambridge, MA 02139, USA.

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