Research ArticleGENETICS

Correction of diverse muscular dystrophy mutations in human engineered heart muscle by single-site genome editing

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Science Advances  31 Jan 2018:
Vol. 4, no. 1, eaap9004
DOI: 10.1126/sciadv.aap9004

Article Information

vol. 4 no. 1

Online ISSN: 
History: 
  • Received for publication September 7, 2017
  • Accepted for publication December 28, 2017

Author Information

  1. Chengzu Long1,2,3,4,*,,
  2. Hui Li1,2,3,*,
  3. Malte Tiburcy5,6,
  4. Cristina Rodriguez-Caycedo1,2,3,
  5. Viktoriia Kyrychenko1,2,3,
  6. Huanyu Zhou1,2,3,
  7. Yu Zhang1,2,3,
  8. Yi-Li Min1,2,3,
  9. John M. Shelton7,
  10. Pradeep P. A. Mammen2,3,7,
  11. Norman Y. Liaw5,
  12. Wolfram-Hubertus Zimmermann5,6,
  13. Rhonda Bassel-Duby1,2,3,
  14. Jay W. Schneider2,3,7 and
  15. Eric N. Olson1,2,3,
  1. 1Department of Molecular Biology, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
  2. 2Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
  3. 3Hamon Center for Regenerative Science and Medicine, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
  4. 4Leon H. Charney Division of Cardiology, New York University School of Medicine, New York, NY 10016, USA.
  5. 5Institute of Pharmacology and Toxicology, University Medical Center, Georg-August-University Göttingen, Göttingen, 37075, Germany.
  6. 6DZHK (German Center for Cardiovascular Research), partner site Göttingen, Göttingen, Germany.
  7. 7Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
  1. Corresponding author. Email: eric.olson{at}utsouthwestern.edu (E.N.O.); chengzu.long{at}nyumc.org (C.L.)
    • * These authors contributed equally to this work.

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