| First Author | Gregorevic P | Year | 2006 |
| Journal | Nat Med | Volume | 12 |
| Issue | 7 | Pages | 787-9 |
| PubMed ID | 16819550 | Mgi Jnum | J:111980 |
| Mgi Id | MGI:3655317 | Doi | 10.1038/nm1439 |
| Citation | Gregorevic P, et al. (2006) rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice. Nat Med 12(7):787-9 |
| abstractText | 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. |
