Author: ["Paul Gregorevic","James M Allen","Elina Minami","Michael J Blankinship","Miki Haraguchi","Leonard Meuse","Eric Finn","Marvin E Adams","Stanley C Froehner","Charles E Murry","Jeffrey S Chamberlain"]
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Abstract
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.Cite this article
Gregorevic, P., Allen, J., Minami, E. et al. rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice. Nat Med 12, 787–789 (2006). https://doi.org/10.1038/nm1439 