NIH R01 · 2025
Gene Therapy for LCA5-Assocated Inherited Retinal Degeneration: Extension to Pediatric Cohorts
PROJECT SUMMARY / ABSTRACT Advances in genetics and genomics have raised hopes for the development of gene-based treatments for inherited retinal diseases (IRDs) which are single-gene defects that cause vision loss. Some of the greatest successes for gene-based treatments has occurred in the most severe subset of IRDs that fall into the rubric of Leber congenital amaurosis (LCA) with vision loss often occurring congenitally. LCA success stories from human clinical trials include those caused by deleterious variants in the genes RPE65, LRAT, CEP290, GUCY2D, and AIPL1. Although these disorders present with severe vision loss in early childhood, the great majority of trials have not included…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.