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

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

Author information

1
Department of Neurology, Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Washington, 1959 NE Pacific Street, Seattle, Washington 98195, USA.

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.

PMID:
16819550
PMCID:
PMC4244883
DOI:
10.1038/nm1439
[Indexed for MEDLINE]
Free PMC Article
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