Preclinical studies for gene therapy of Duchenne muscular dystrophy

Guy L. Odom, Glen B. Banks, Brian R. Schultz, Paul Gregorevic, Jeffrey S Chamberlain

Research output: Contribution to journalReview ArticleResearchpeer-review

9 Citations (Scopus)

Abstract

The muscular dystrophies are a diverse group of genetic disorders without an effective treatment. Because they are caused by mutations in various genes, the most direct way to treat them involves correcting the underlying gene defect (ie, gene therapy). Such a gene therapy approach involves delivering a therapeutic gene cassette to essentially all the muscles of the body in a safe and efficacious manner. The authors describe gene delivery methods using vectors derived from adeno-associated virus that are showing great promise in preclinical studies for treatment of Duchenne muscular dystrophy. It is hoped that variations on these methods might be applicable for most, if not all, of the different types of muscular dystrophy.

Original languageEnglish
Pages (from-to)1149-1157
Number of pages9
JournalJournal of Child Neurology
Volume25
Issue number9
DOIs
Publication statusPublished - Sep 2010
Externally publishedYes

Keywords

  • adeno-associated virus
  • Duchenne muscular dystrophy
  • dystrophin
  • gene therapy
  • micro-dystrophin

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