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In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

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Science  22 Jan 2016:
Vol. 351, Issue 6271, pp. 403-407
DOI: 10.1126/science.aad5143

Article Information

vol. 351 no. 6271 403-407

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History: 
  • Received for publication September 24, 2015
  • Accepted for publication December 7, 2015

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Author Information

  1. Christopher E. Nelson1,2,
  2. Chady H. Hakim3,
  3. David G. Ousterout1,2,
  4. Pratiksha I. Thakore1,2,
  5. Eirik A. Moreb1,2,
  6. Ruth M. Castellanos Rivera4,
  7. Sarina Madhavan1,2,
  8. Xiufang Pan3,
  9. F. Ann Ran5,6,
  10. Winston X. Yan5,7,8,
  11. Aravind Asokan4,
  12. Feng Zhang5,9,10,11,
  13. Dongsheng Duan3,12,
  14. Charles A. Gersbach1,2,13,*
  1. 1Department of Biomedical Engineering, Duke University, Durham, NC, USA.
  2. 2Center for Genomic and Computational Biology, Duke University, Durham, NC, USA.
  3. 3Department of Molecular Microbiology and Immunology, University of Missouri, Columbia, MO, USA.
  4. 4Gene Therapy Center, Departments of Genetics, Biochemistry and Biophysics, School of Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
  5. 5Broad Institute of MIT and Harvard, Cambridge, MA, USA.
  6. 6Society of Fellows, Harvard University, Cambridge, MA, USA.
  7. 7Graduate Program in Biophysics, Harvard Medical School, Boston, MA, USA.
  8. 8Harvard–MIT Division of Health Sciences and Technology, Harvard Medical School, Boston, MA, USA.
  9. 9McGovern Institute for Brain Research, Massachusetts Institute of Technology, Cambridge, MA, USA.
  10. 10Department of Brain and Cognitive Sciences, Massachusetts Institute of Technology, Cambridge, MA, USA.
  11. 11Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA, USA.
  12. 12Department of Neurology, University of Missouri, Columbia, MO, USA.
  13. 13Department of Orthopaedic Surgery, Duke University Medical Center, Durham, NC, USA.
  1. *Corresponding author. E-mail: charles.gersbach{at}duke.edu

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Article usage: December 2015 to November 2018

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