Long-term benefit of adeno-associated virus/antisense-mediated exon skipping in dystrophic mice

Hum Gene Ther. 2008 Jun;19(6):601-8. doi: 10.1089/hum.2008.012.


Many mutations and deletions in the dystrophin gene, responsible for Duchenne muscular dystrophy (DMD), can be corrected at the posttranscriptional level by skipping specific exons. Here we show that long-term benefit can be obtained in the dystrophic mouse model through the use of adeno-associated viral vectors expressing antisense sequences: persistent exon skipping, dystrophin rescue, and functional benefit were observed 74 weeks after a single systemic administration. The therapeutic benefit was sufficient to preserve the muscle integrity of mice up to old age. These results indicate a possible long-term gene therapy treatment of the DMD pathology.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Dependovirus*
  • Disease Models, Animal
  • Dystrophin / genetics*
  • Dystrophin / metabolism
  • Exons
  • Female
  • Genetic Therapy / methods*
  • Genetic Vectors*
  • Mice
  • Mice, Inbred mdx
  • Muscle, Skeletal / metabolism
  • Muscle, Skeletal / pathology
  • Muscle, Skeletal / physiopathology
  • Muscular Dystrophy, Duchenne / therapy*
  • RNA, Antisense / genetics*


  • Dystrophin
  • RNA, Antisense