Research ArticleMuscular Dystrophy

Dantrolene Enhances Antisense-Mediated Exon Skipping in Human and Mouse Models of Duchenne Muscular Dystrophy

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Science Translational Medicine  12 Dec 2012:
Vol. 4, Issue 164, pp. 164ra160
DOI: 10.1126/scitranslmed.3005054

Article Information

vol. 4 no. 164 164ra160

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History: 
  • Received for publication September 28, 2012
  • Accepted for publication November 19, 2012

Author Information

  1. Genevieve C. Kendall1,2,3,*,
  2. Ekaterina I. Mokhonova3,4,*,
  3. Miriana Moran3,5,
  4. Natalia E. Sejbuk3,5,
  5. Derek W. Wang3,5,
  6. Oscar Silva5,
  7. Richard T. Wang1,3,
  8. Leonel Martinez3,4,
  9. Qi L. Lu6,
  10. Robert Damoiseaux7,
  11. Melissa J. Spencer3,4,*,
  12. Stanley F. Nelson1,2,3,8,*, and
  13. M. Carrie Miceli2,3,5,*,
  1. 1Department of Human Genetics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA 90095, USA.
  2. 2Molecular Biology Institute, University of California, Los Angeles, Los Angeles, CA 90095, USA.
  3. 3Center for Duchenne Muscular Dystrophy, University of California, Los Angeles, Los Angeles, CA 90095, USA.
  4. 4Department of Neurology, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA 90095, USA.
  5. 5Department of Microbiology, Immunology, and Molecular Genetics, David Geffen School of Medicine and College of Letters and Sciences, University of California, Los Angeles, Los Angeles, CA 90095, USA.
  6. 6Department of Neurology, McColl-Lockwood Laboratory of Muscular Dystrophy Research, Carolinas Medical Center, Charlotte, NC 28231, USA.
  7. 7California Nanosciences Institute, University of California, Los Angeles, Los Angeles, CA 90095, USA.
  8. 8Department of Pathology and Laboratory Medicine, University of California, Los Angeles, Los Angeles, CA 90095, USA.
  1. To whom correspondence should be addressed. E-mail: cmiceli{at}ucla.edu (M.C.M.); snelson{at}ucla.edu (S.F.N.)
  • * These authors contributed equally to this work.

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