Research ArticleGENETICS

CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells

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Science Advances  06 Mar 2019:
Vol. 5, no. 3, eaav4324
DOI: 10.1126/sciadv.aav4324

Article Information

vol. 5 no. 3

Online ISSN: 
History: 
  • Received for publication September 14, 2018
  • Accepted for publication January 28, 2019
  • .

Author Information

  1. Yi-Li Min1,2,
  2. Hui Li1,2,
  3. Cristina Rodriguez-Caycedo1,2,
  4. Alex A. Mireault1,2,
  5. Jian Huang3,
  6. John M. Shelton3,
  7. John R. McAnally1,2,
  8. Leonela Amoasii1,2,4,
  9. Pradeep P. A. Mammen2,3,
  10. Rhonda Bassel-Duby1,2 and
  11. Eric N. Olson1,2,*
  1. 1Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  2. 2Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  3. 3Department of Internal Medicine, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  4. 4Exonics Therapeutics, 490 Arsenal Way, Watertown, MA 02472, USA.
  1. *Corresponding author. Email: eric.olson{at}utsouthwestern.edu

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