Article
Research Models and Gene Augmentation Therapy for CRB1 Retinal Dystrophies
14 Aug 2020
Abstract excerpt
Retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA) are inherited degenerative retinal dystrophies with vision loss that ultimately lead to blindness. Several genes have been shown to be involved in early onset retinal dystrophies, including CRB1 and RPE65. Gene therapy recently became available for young RP patients with variations in the RPE65 gene. Current research programs test adeno-associated...
Topics
Join the communities discussing this publication.