NIH R01 · 2024
Photoreceptor Disease in Inherited Retinal Degenerations
PROJECT SUMMARY / ABSTRACT Loss of eyesight is regarded among the worst possible diseases by most Americans. Despite being rare causes of vision loss, inherited retinal diseases (IRDs) are molecularly simple single-gene defects, and advances in genetics and genomics have raised hopes for the development of gene-based treatments. For most IRDs, the natural history of disease involves progressive vision loss and successful interventions must demonstrate a clinically meaningful slowing of the natural progression. Many good metrics have been developed to measure vision loss but the continued lack of approved IRD treatments strongly suggest better outcome measures are still needed. The long-term…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.