Gene Therapy for Rdh12-Associated Retinal Diseases Helps to Delay Retinal Degeneration and Vision Loss.

ABSTRACT:

Purpose

The aim of study was to establish Rdh12-associated inherited retinal disease (Rdh12-IRD) mouse model and to identify the best timepoint for gene therapy.

Methods

We induced retinal degeneration in Rdh12^-/- mice using a bright light. We clarified the establishment of Rdh12-IRD mouse model by analyzing the thickness of retinal layers and electroretinography (ERG). Rdh12-IRD mice received a subretinal injection of adeno-associated virus 2/8-packaged Rdh12 cDNA for treatment. We evaluated the visual function and retinal structure in the treated and untreated eyes to identify the best timepoint for gene therapy.

Results

Rdh12-IRD mice showed significant differences in ERG amplitudes and photoreceptor survi

SUBMITTER: Bian J 

PROVIDER: S-EPMC8380142 | biostudies-literature | 2021

REPOSITORIES: biostudies-literature