rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice.
Nat Med
; 12(7): 787-9, 2006 Jul.
Article
en En
| MEDLINE
| ID: mdl-16819550
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.
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Músculos Respiratorios
/
Distrofina
/
Técnicas de Transferencia de Gen
/
Dependovirus
/
Músculo Esquelético
/
Distrofia Muscular Animal
Límite:
Animals
Idioma:
En
Revista:
Nat Med
Asunto de la revista:
BIOLOGIA MOLECULAR
/
MEDICINA
Año:
2006
Tipo del documento:
Article
País de afiliación:
Estados Unidos