FocusDuchenne Muscular Dystrophy

Exon-Skipping Therapy: A Roadblock, Detour, or Bump in the Road?

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Science Translational Medicine  02 Apr 2014:
Vol. 6, Issue 230, pp. 230fs14
DOI: 10.1126/scitranslmed.3008873

Article Information

vol. 6 no. 230 230fs14

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Author Information

  1. Eric P. Hoffman1,* and
  2. Elizabeth M. McNally2
  1. 1Center for Genetic Medicine Research, Children’s National Medical Center, 111 Michigan Avenue, NW, Washington, DC 20010, USA.
  2. 2Departments of Medicine and Human Genetics, Institute for Cardiovascular Research, University of Chicago Medical Center, 5841 S. Maryland Avenue, MC 6088, Chicago, IL 60637, USA.
  1. *Corresponding author. E-mail: ehoffman{at}childrensnational.org

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