Versatility of AAV vectors for retinal gene transfer.
Vision Res
; 48(3): 353-9, 2008 Feb.
Article
en En
| MEDLINE
| ID: mdl-17923143
ABSTRACT
Gene therapy represents a promising therapeutic option for many inherited and acquired retinal diseases. Recombinant adeno-associated viral vectors (AAV) are the most efficient tools to transfer genes in vivo to the retina. The recent identification of dozens of novel AAV serotypes enormously expands on the versatility of AAV as vector system for in vivo somatic gene transfer. The results from the forthcoming trials with AAV in the retina of patients with Leber Congenital Amaurosis will be critical for the rapid development of AAV-based therapeutics for retinal diseases.
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Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Enfermedades de la Retina
/
Enfermedades Hereditarias del Ojo
/
Técnicas de Transferencia de Gen
/
Dependovirus
/
Vectores Genéticos
Límite:
Animals
/
Humans
Idioma:
En
Revista:
Vision Res
Año:
2008
Tipo del documento:
Article
País de afiliación:
Italia