Metabolic plasticity in a Pde6b^STOP/STOP retinitis pigmentosa mouse model following rescue.

ABSTRACT:

Objective

Retinitis pigmentosa (RP) is a hereditary retinal disease characterized by progressive photoreceptor degeneration, leading to vision loss. The best hope for a cure for RP lies in gene therapy. However, given that RP patients are most often diagnosed in the midst of ongoing photoreceptor degeneration, it is unknown how the retinal proteome changes as RP disease progresses, and which changes can be prevented, halted, or reversed by gene therapy.

Methods

Here, we used a Pde6b-deficient RP gene therapy mouse model and performed untargeted proteomic analysis to identify changes in protein expression during degeneration and after treatment.

Results

We demonstrated that Pde6b gene restoration led to a novel form of homeostatic plasticity in rod phototransduction wh

SUBMITTER: Ayten M 

PROVIDER: S-EPMC11362769 | biostudies-literature | 2024 Jul

REPOSITORIES: biostudies-literature