NIH R01 · 2025
Retinal Pigment Epithelium Secreted Matrix Metalloproteinase 2 as a Novel Target in Choroideremia
PROJECT SUMMARY: Choroideremia is a blinding inherited retinal disease in which retinal pigment epithelial (RPE) cells contribute significantly to chorioretinal degeneration, but the underlying molecular mechanisms remain largely unclear. A critical barrier to progress in developing therapies is understanding how RPE cells contribute to death of neighboring tissues in choroideremia. Our long-term goal is to determine how RPE dysfunction contributes to chorioretinal degeneration and to develop novel therapies. Choroideremia is caused by mutations in CHM, encoding Rab escort protein 1, which facilitates prenylation of Rab GTPases (Rabs). Prenylation is required for Rabs to associate with…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.