Long-term benefit of adeno-associated virus/antisense-mediated exon skipping in dystrophic mice.
Hum Gene Ther
; 19(6): 601-8, 2008 Jun.
Article
em En
| MEDLINE
| ID: mdl-18500943
ABSTRACT
Many mutations and deletions in the dystrophin gene, responsible for Duchenne muscular dystrophy (DMD), can be corrected at the posttranscriptional level by skipping specific exons. Here we show that long-term benefit can be obtained in the dystrophic mouse model through the use of adeno-associated viral vectors expressing antisense sequences persistent exon skipping, dystrophin rescue, and functional benefit were observed 74 weeks after a single systemic administration. The therapeutic benefit was sufficient to preserve the muscle integrity of mice up to old age. These results indicate a possible long-term gene therapy treatment of the DMD pathology.
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Terapia Genética
/
Distrofina
/
RNA Antissenso
/
Dependovirus
/
Distrofia Muscular de Duchenne
/
Vetores Genéticos
Tipo de estudo:
Risk_factors_studies
Limite:
Animals
Idioma:
En
Ano de publicação:
2008
Tipo de documento:
Article