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Research ArticleMuscular Dystrophy
Dantrolene Enhances Antisense-Mediated Exon Skipping in Human and Mouse Models of Duchenne Muscular Dystrophy
- Genevieve C. Kendall1,2,3,*,
- Ekaterina I. Mokhonova3,4,*,
- Miriana Moran3,5,
- Natalia E. Sejbuk3,5,
- Derek W. Wang3,5,
- Oscar Silva5,
- Richard T. Wang1,3,
- Leonel Martinez3,4,
- Qi L. Lu6,
- Robert Damoiseaux7,
- Melissa J. Spencer3,4,*,
- Stanley F. Nelson1,2,3,8,*,† and
- M. Carrie Miceli2,3,5,*,†
- 1Department of Human Genetics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA 90095, USA.
- 2Molecular Biology Institute, University of California, Los Angeles, Los Angeles, CA 90095, USA.
- 3Center for Duchenne Muscular Dystrophy, University of California, Los Angeles, Los Angeles, CA 90095, USA.
- 4Department of Neurology, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA 90095, USA.
- 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.
- 6Department of Neurology, McColl-Lockwood Laboratory of Muscular Dystrophy Research, Carolinas Medical Center, Charlotte, NC 28231, USA.
- 7California Nanosciences Institute, University of California, Los Angeles, Los Angeles, CA 90095, USA.
- 8Department of Pathology and Laboratory Medicine, University of California, Los Angeles, Los Angeles, CA 90095, USA.
- ↵†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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Science Translational Medicine 12 Dec 2012:
Vol. 4, Issue 164, pp. 164ra160
DOI: 10.1126/scitranslmed.3005054
Vol. 4, Issue 164, pp. 164ra160
DOI: 10.1126/scitranslmed.3005054
Genevieve C. Kendall
1Department of Human Genetics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA 90095, USA.
2Molecular Biology Institute, University of California, Los Angeles, Los Angeles, CA 90095, USA.
3Center for Duchenne Muscular Dystrophy, University of California, Los Angeles, Los Angeles, CA 90095, USA.
Ekaterina I. Mokhonova
3Center for Duchenne Muscular Dystrophy, University of California, Los Angeles, Los Angeles, CA 90095, USA.
4Department of Neurology, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA 90095, USA.
Miriana Moran
3Center for Duchenne Muscular Dystrophy, University of California, Los Angeles, Los Angeles, CA 90095, USA.
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.
Natalia E. Sejbuk
3Center for Duchenne Muscular Dystrophy, University of California, Los Angeles, Los Angeles, CA 90095, USA.
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.
Derek W. Wang
3Center for Duchenne Muscular Dystrophy, University of California, Los Angeles, Los Angeles, CA 90095, USA.
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.
Oscar Silva
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.
Richard T. Wang
1Department of Human Genetics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA 90095, USA.
3Center for Duchenne Muscular Dystrophy, University of California, Los Angeles, Los Angeles, CA 90095, USA.
Leonel Martinez
3Center for Duchenne Muscular Dystrophy, University of California, Los Angeles, Los Angeles, CA 90095, USA.
4Department of Neurology, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA 90095, USA.
Qi L. Lu
6Department of Neurology, McColl-Lockwood Laboratory of Muscular Dystrophy Research, Carolinas Medical Center, Charlotte, NC 28231, USA.
Robert Damoiseaux
7California Nanosciences Institute, University of California, Los Angeles, Los Angeles, CA 90095, USA.
Melissa J. Spencer
3Center for Duchenne Muscular Dystrophy, University of California, Los Angeles, Los Angeles, CA 90095, USA.
4Department of Neurology, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA 90095, USA.
Stanley F. Nelson
1Department of Human Genetics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA 90095, USA.
2Molecular Biology Institute, University of California, Los Angeles, Los Angeles, CA 90095, USA.
3Center for Duchenne Muscular Dystrophy, University of California, Los Angeles, Los Angeles, CA 90095, USA.
8Department of Pathology and Laboratory Medicine, University of California, Los Angeles, Los Angeles, CA 90095, USA.
M. Carrie Miceli
2Molecular Biology Institute, University of California, Los Angeles, Los Angeles, CA 90095, USA.
3Center for Duchenne Muscular Dystrophy, University of California, Los Angeles, Los Angeles, CA 90095, USA.
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.
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