Gene Therapy
Inherited retinal degenerations result in progressive, irreversible blindness. Gene therapy provides healthy copies of a missing or mutated gene to improve visual function.
We are currently developing adeno-associated viral (AAV) gene therapy for gyrate atrophy, a retinal disease caused by mutations in ornithine aminotransferase (OAT).
Cell Therapy
Rod and cone photoreceptors are highly specialized cells that convert light into electrical signals. These cells cannot regenerate in humans, and thus photoreceptor cell death currently leads to permanent blindness.
We study retinal development and are investigating cell-based therapies for photoreceptor regeneration, in the hope of restoring vision in patients with late-stage retinal disease from any underlying cause.
We are grateful for generous funding and support from:
Commonwealth of Pennsylvania Montague Investigator Award McGaughey Family