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

Genevieve C. Kendall; Ekaterina I. Mokhonova; Miriana Moran; Natalia E. Sejbuk; Derek W. Wang; Oscar Silva; Richard T. Wang; Leonel Martinez; Qi L. Lu; Robert Damoiseaux; Melissa J. Spencer; Stanley F. Nelson; M. Carrie Miceli

doi:10.1126/scitranslmed.3005054

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

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.

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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.

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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.

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Natalia E. Sejbuk

3Center for Duchenne Muscular Dystrophy, University of California, Los Angeles, Los Angeles, CA 90095, USA.

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Derek W. Wang

3Center for Duchenne Muscular Dystrophy, University of California, Los Angeles, Los Angeles, CA 90095, USA.

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Oscar Silva

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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.

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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.

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Qi L. Lu

6Department of Neurology, McColl-Lockwood Laboratory of Muscular Dystrophy Research, Carolinas Medical Center, Charlotte, NC 28231, USA.

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Robert Damoiseaux

7California Nanosciences Institute, University of California, Los Angeles, Los Angeles, CA 90095, USA.

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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.

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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.

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For correspondence: cmiceli@ucla.edu snelson@ucla.edu

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.

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For correspondence: cmiceli@ucla.edu snelson@ucla.edu

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

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