Mutation-Independent Gene Therapies for Rod-Cone Dystrophies.
Adv Exp Med Biol
; 1074: 75-81, 2018.
Article
en En
| MEDLINE
| ID: mdl-29721930
ABSTRACT
The clinical success of gene replacement therapies in recent years has served as a proof of concept for the treatment of inherited retinal degenerations using adeno-associated virus (AAV) as viral vector. However, inherited retinal degenerative diseases showcase a broad genetic and mechanistic heterogeneity, challenging the development of mutation-specific therapies for each specific mutation. Mutation-independent approaches must be developed to slow down retinal degeneration regardless of the underlying genetic mutation and onset of the disease. New understanding of cell death mechanisms in rod-cone dystrophies have led to promising rescue of photoreceptor cell death by virally mediating expression of anti-apoptotic factors and secretion of retinal neurotrophic factors. Optogenetic therapies are also able to restore light sensitivities in blind retinas.
Palabras clave
Texto completo:
1
Banco de datos:
MEDLINE
Asunto principal:
Terapia Genética
/
Optogenética
/
Distrofias de Conos y Bastones
/
Vectores Genéticos
Límite:
Humans
Idioma:
En
Año:
2018
Tipo del documento:
Article