Journal
NATURE MEDICINE
Volume 12, Issue 7, Pages 787-789Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/nm1439
Keywords
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Funding
- NIAMS NIH HHS [R37 AR040864] Funding Source: Medline
- NINDS NIH HHS [P01 NS046788] Funding Source: Medline
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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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