RPE-Directed Gene Therapy Improves Mitochondrial Function in Murine Dry AMD Models.

ABSTRACT: Age-related macular degeneration (AMD) is the most common cause of blindness in the aged population. However, to date there is no effective treatment for the dry form of the disease, representing 85-90% of cases. AMD is an immensely complex disease which affects, amongst others, both retinal pigment epithelium (RPE) and photoreceptor cells and leads to the progressive loss of central vision. Mitochondrial dysfunction in both RPE and photoreceptor cells is emerging as a key player in the disease. There are indications that during disease progression, the RPE is first impaired and RPE dysfunction in turn leads to subsequent photoreceptor cell degeneration; however, the exact sequence of events has not as yet been fully determined. We recently showed that AAV delivery of an optimised NADH-ubi

SUBMITTER: Millington-Ward S 

PROVIDER: S-EPMC9968062 | biostudies-literature | 2023 Feb

REPOSITORIES: biostudies-literature