Restoration of cone vision in a mouse model of achromatopsia.
Nat Med
; 13(6): 685-7, 2007 Jun.
Article
en En
| MEDLINE
| ID: mdl-17515894
ABSTRACT
Loss of cone function in the central retina is a pivotal event in the development of severe vision impairment for many prevalent blinding diseases. Complete achromatopsia is a genetic defect resulting in cone vision loss in 1 in 30,000 individuals. Using adeno-associated virus (AAV) gene therapy, we show that it is possible to target cones and rescue both the cone-mediated electroretinogram response and visual acuity in the Gnat2 ( cpfl3 ) mouse model of achromatopsia.
Texto completo:
1
Colección:
01-internacional
Banco de datos:
MEDLINE
Asunto principal:
Terapia Genética
/
Defectos de la Visión Cromática
/
Células Fotorreceptoras Retinianas Conos
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Modelos Animales de Enfermedad
Límite:
Animals
Idioma:
En
Revista:
Nat Med
Asunto de la revista:
BIOLOGIA MOLECULAR
/
MEDICINA
Año:
2007
Tipo del documento:
Article
País de afiliación:
Estados Unidos