Nature Medicine
- 12, 787 - 789 (2006)
Published online: 2 July 2006; | doi:10.1038/nm1439
rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic micePaul Gregorevic1, James M Allen1, 2, Elina Minami3, Michael J Blankinship1, Miki Haraguchi1, Leonard Meuse1, Eric Finn1, Marvin E Adams4, Stanley C Froehner4, Charles E Murry3 & Jeffrey S Chamberlain1, 2, 51
Department of Neurology, Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Washington, 1959 NE Pacific Street, Seattle, Washington 98195, USA. 2
Department of Medicine, University of Washington, 1959 NE Pacific Street, Seattle, Washington 98195, USA. 3
Department of Pathology, and Center for Cardiovascular Biology and Regenerative Medicine, University of Washington, 1959 NE Pacific Street, Seattle, Washington 98195, USA. 4
Department of Physiology and Biophysics, University of Washington, 1959 NE Pacific Street, Seattle, Washington 98195, USA. 5
Department of Biochemistry, University of Washington, 1959 NE Pacific Street, Seattle, Washington 98195, USA.
Correspondence should be addressed to Jeffrey S Chamberlain jsc5@u.washington.edu 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.
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