Research ArticleMuscular Dystrophy

Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy

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Science Translational Medicine  29 Nov 2017:
Vol. 9, Issue 418, eaan8081
DOI: 10.1126/scitranslmed.aan8081

Article Information

vol. 9 no. 418

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  • Received for publication May 22, 2017
  • Accepted for publication October 18, 2017

Author Information

  1. Leonela Amoasii1,*,
  2. Chengzu Long1,*,,
  3. Hui Li1,
  4. Alex A. Mireault1,
  5. John M. Shelton2,
  6. Efrain Sanchez-Ortiz1,
  7. John R. McAnally1,
  8. Samadrita Bhattacharyya1,
  9. Florian Schmidt3,
  10. Dirk Grimm3,
  11. Stephen D. Hauschka4,
  12. Rhonda Bassel-Duby1 and
  13. Eric N. Olson1,
  1. 1Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  2. 2Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
  3. 3Heidelberg University Hospital, Center for Infectious Diseases, Virology, Cluster of Excellence Cell Networks, DZIF partner, BioQuant Center, Heidelberg D-69120, Germany.
  4. 4Department of Biochemistry, University of Washington, Seattle, WA 98195, USA.
  1. Corresponding author. Email: eric.olson{at}
    • * These authors contributed equally to this work.

    • Present address: Leon H. Charney Division of Cardiology, New York University School of Medicine, New York, NY 10016, USA.


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