Gene replacement therapy restores vision in mice

Dr. Visvanathan Ramamurthy, a researcher at the West Virginia University Eye Institute and the WVU Center for Neuroscience, and WVU MD/PhD student Cristy Ku have recently demonstrated that gene replacement therapy can restore sight in animal models. Using viral mediated gene replacement therapy, these researchers were able to restore vision in mice up to two months after a single treatment. Hopefully, this pre-clinical model can be used to treat leber congenital amaurosis (LCA), a rare genetic disease that leads to photoreceptor cell death and ensuing blindness   in children. This breakthrough by Ramamurthy and Ku may lead to wider utilization of gene therapy to treat many eye diseases in the future.