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.
journal_name
Nat Medjournal_title
Nature medicineauthors
Gregorevic P,Allen JM,Minami E,Blankinship MJ,Haraguchi M,Meuse L,Finn E,Adams ME,Froehner SC,Murry CE,Chamberlain JSdoi
10.1038/nm1439subject
Has Abstractpub_date
2006-07-01 00:00:00pages
787-9issue
7eissn
1078-8956issn
1546-170Xpii
nm1439journal_volume
12pub_type
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