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Gene therapy for Duchenne muscular dystrophy

14 Aug 2012

Abstract excerpt

PURPOSE OF REVIEW: Duchenne muscular dystrophy is a severe neuromuscular disorder for which there is currently no cure. Years of research have come to fruition during the past 18 months with publications on clinical trials for several gene therapy approaches for Duchenne muscular dystrophy. This review covers the present status of these approaches. RECENT FINDINGS: The exon skipping approach is most advanced in...

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PMID: 22892952
DOI: 10.1097/wco.0b013e328357b0be

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