rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice

Nat Med. 2006 Jul;12(7):787-9. doi: 10.1038/nm1439. Epub 2006 Jul 2.


Mice carrying mutations in both the dystrophin and utrophin genes die prematurely as a consequence of severe muscular dystrophy. Here, we show that intravascular administration of recombinant adeno-associated viral (rAAV) vectors carrying a microdystrophin gene restores expression of dystrophin in the respiratory, cardiac and limb musculature of these mice, considerably reducing skeletal muscle pathology and extending lifespan. These findings suggest rAAV vector-mediated systemic gene transfer may be useful for treatment of serious neuromuscular disorders such as Duchenne muscular dystrophy.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Dependovirus / genetics*
  • Dystrophin / genetics*
  • Dystrophin / therapeutic use
  • Gene Transfer Techniques*
  • Genetic Vectors
  • Longevity
  • Mice
  • Muscle Fibers, Skeletal / physiology
  • Muscle, Skeletal / physiopathology*
  • Muscular Dystrophy, Animal / genetics
  • Muscular Dystrophy, Animal / physiopathology
  • Muscular Dystrophy, Animal / therapy*
  • Respiratory Muscles / physiopathology*


  • Dystrophin