version of RPE65 gene therapy

RPE65 gene therapy is a treatment aimed at restoring vision in individuals with specific hereditary retinal diseases caused by mutations in the RPE65 gene. This gene is crucial for the functioning of retinal cells that convert light into visual signals. In this therapy, a modified virus is used to deliver a healthy copy of the RPE65 gene directly into the retina. By providing the correct genetic instructions, the therapy helps these retinal cells regain their ability to detect light, potentially improving vision for patients with conditions like Leber congenital amaurosis and retinitis pigmentosa.