NIH R01 · 2024
Models for Therapy of Hereditary Retinal Degeneration
Inherited retinal diseases (IRDs) such as retinitis pigmentosa, Leber congenital amaurosis, cone-rod dystrophy, and Best macular dystrophy are devastating blinding diseases in people. While mutations in nearly 270 genes have been associated with different forms of IRDs to date, characterization of disease mechanisms and identification of therapeutic targets for many of these IRDs are yet to be developed. Encouraging clinical successes with gene replacement therapy have emerged in recent years for several forms of IRD in man, and some of these treatments have resulted from proof of principle studies carried out in dog models by our research group. At the Retinal Disease Study Facility of the…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.