Targeting miR-181a/b in retinitis pigmentosa: implications for disease progression and therapy.

ABSTRACT:

Background

Retinitis pigmentosa (RP) is a genetically heterogeneous group of degenerative disorders causing progressive vision loss due to photoreceptor death. RP affects other retinal cells, including the retinal pigment epithelium (RPE). MicroRNAs (miRs) are implicated in RP pathogenesis, and downregulating miR-181a/b has shown therapeutic benefit in RP mouse models by improving mitochondrial function. This study investigates the expression profile of miR-181a/b in RPE cells and the neural retina during RP disease progression. We also evaluate how miR-181a/b downregulation, by knocking out miR-181a/b-1 cluster in RPE cells, confers therapeutic efficacy in an RP mouse model and explore the mechanisms underlying this process.

Results

Our findings reveal distinct expression p

SUBMITTER: Costa BLD 

PROVIDER: S-EPMC11110387 | biostudies-literature | 2024 May

REPOSITORIES: biostudies-literature