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J Child Neurol. 2010 Sep;25(9):1145-8. doi: 10.1177/0883073810371005. Epub 2010 May 24.

Molecular therapeutic strategies targeting Duchenne muscular dystrophy.

Author information

  • 1Center for Gene Therapy, Nationwide Children's Hospital, Department of Pediatrics, The Ohio State University, Columbus, OH 43205, USA. jerry.mendell@nationwidechildrens.org

Abstract

Since the discovery of the gene for Duchenne muscular dystrophy more than 20 years ago, scientists have worked to apply molecular principles for restoration of the dystrophin protein and correction of the underlying physiologic defect that predisposes muscle fibers to injury. Recent studies provide realistic hope that molecular therapies may help patients who have this disorder. At present, only corticosteroids can improve walking ability and increase quality of life for boys with this disease. The results are modest and encumbered by side effects. The authors review 3 molecular therapeutic approaches that have been introduced into the clinic: (1) gene replacement therapy, (2) mutation suppression, and (3) exon skipping.

PMID:
20498331
[PubMed - indexed for MEDLINE]
PMCID:
PMC3674570
Free PMC Article
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