Gene Therapy for Inherited Retinal Diseases
Delivering gene therapy to the RPE
The delivery of a functional gene to the retinal pigment epithelium (RPE) has been achieved in patients with certain types of Leber congenital amaurosis (LCA) and choroideremia. In some, an improvement in retinal function as well as vision has been demonstrated. Clinical trials are ongoing for other retinal dystrophies such as Stargardt disease, X-linked juvenile retinoschisis and Usher syndrome type 1B, with results expected in the next few years....