Developments in gene therapy for muscular dystrophy

Dennis Hartigan-O'Connor, Jeffrey S. Chamberlain

Research output: Contribution to journalArticlepeer-review

61 Scopus citations


Gene therapy for muscular dystrophy (MD) presents significant challenges, including the large amount of muscle tissue in the body, the large size of many genes defective in different muscular dystrophies, and the possibility of a host immune response against the therapeutic gene. Overcoming these challenges requires the development and delivery of suitable gene transfer vectors. Encouraging progress has been made in modifying adenovirus (Ad) vectors to reduce immune response and increase capacity. Recently developed gutted Ad vectors can deliver full-length dystrophin cDNA expression vectors to muscle tissue. Using muscle-specific promoters to drive dystrophin expression, a strong immune response has not been observed in mdx mice. Adeno-associated virus (AAV) vectors can deliver small genes to muscle without provocation of a significant immune response, which should allow long-term expression of several MD genes. AAV vectors have also been used to deliver sarcoglycan genes to entire muscle groups. These advances and others reviewed here suggest that barriers to gene therapy for MD are surmountable. (C) 2000 Willey-Liss, Inc.

Original languageEnglish (US)
Pages (from-to)223-238
Number of pages16
JournalMicroscopy Research and Technique
Issue number3-4
StatePublished - 2000
Externally publishedYes


  • AAV
  • Adenovirus
  • Duchenne
  • Dystrophin
  • Gutless
  • Gutted
  • Helper- dependent
  • Lentivirus
  • Limb-girdle
  • Vector

ASJC Scopus subject areas

  • Agricultural and Biological Sciences(all)
  • Anatomy
  • Instrumentation


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