Vision on gyrate atrophy: why treat the eye?
EMBO Mol Med
; 16(1): 4-7, 2024 Jan.
Article
en En
| MEDLINE
| ID: mdl-38177529
ABSTRACT
In the April issue of this Journal, Boffa and coworkers put forward a new therapeutic approach for Gyrate Atrophy of the Choroid and Retina (GACR; OMIM 258870) (Boffa et al, 2023). The authors propose to apply gene therapy to the liver for GACR, a metabolic disease primarily affecting eyesight due to retinal degeneration. Their vision is enthusiastically supported by a News and Views comment in the same issue (Seker Yilmaz and Gissen, 2023). However, based on disease pathology, patient's needs, ethical considerations, therapeutic developmental time lines, and current state of the art of gene therapy for liver and eye, we have a different view on this issue We argue below that local treatment of the eye is the preferred option for GACR.
Texto completo:
1
Base de datos:
MEDLINE
Asunto principal:
Degeneración Retiniana
/
Atrofia Girata
Límite:
Humans
Idioma:
En
Revista:
EMBO Mol Med
/
EMBO molecular medicine (Online)
Asunto de la revista:
BIOLOGIA MOLECULAR
Año:
2024
Tipo del documento:
Article
País de afiliación:
Países Bajos