Our Research

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). This work is supported by our non-profit partner Conquering Gyrate Atrophy; check out the Conquering Gyrate Atrophy website for additional details and updates.

 

A green, maroon, and black figure is visible within a photo taken by a powerful microscope, depicting scaled genetic material up close.

 

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.

 

A vibrant scientific photo including maroon, lime green, and black depiction of cells pictured by a powerful microscope.