Register to attend a free NIH webinar. Attendees will learn about a new gene therapy method for treating Oculocutaneous Albinism (OCA), a genetically heterogeneous disorder caused by a mutation in the Tyrosinase (TYR) gene, which is responsible for the production of melanin (brown-black) pigment in the hair, skin and eyes. This mutation causes a lack of melanin in eye tissues, such as the retinal pigment epithelium (RPE), resulting in abnormal eye development and impaired vision.
Drs. Brian P. Brooks and Aman George, of the National Eye Institute’s (NEI’s) Pediatric, Developmental & Genetic Ophthalmology Section, developed a novel gene therapy treatment for OCA that introduces a corrected copy of the human TYR gene to the eye’s RPE layer and induces melanin production, thereby reversing OCA’s effects on the eye. The NEI is looking for a licensee or co-development partner to commercially develop this technology for the OCA patient population.
