Gene Therapy for Retinal Degeneration.
Cell
; 173(1): 5, 2018 03 22.
Article
en En
| MEDLINE
| ID: mdl-29570997
ABSTRACT
Biallelic mutations in the RPE65 gene are associated with inherited retinal degenerations/dystrophies (IRD) and disrupt the visual cycle, leading to loss of vision. A new adenoviral vector-based gene therapy surgically delivered to retinal cells provides normal human RPE65 protein that can restore the visual cycle and some vision. To view this Bench to Bedside, open or download the PDF.
Texto completo:
1
Colección:
01-internacional
Banco de datos:
MEDLINE
Asunto principal:
Degeneración Retiniana
Límite:
Humans
Idioma:
En
Revista:
Cell
Año:
2018
Tipo del documento:
Article