MORGANTOWN, W.Va. – Visvanathan Ramamurthy, Ph.D., researcher at the West Virginia University Eye Institute and the WVU Center for Neuroscience, and WVU MD/PhD student Cristy Ku recently demonstrated that gene replacement therapy restores sight in animal models. If successful in long-term studies, their research may create vision restoration options for childhood blindness patients.
Dr. Ramamurthy and Ku showed restoration of vision in mice up to two months after a single treatment using the latest advances in viral mediated gene replacement therapy. The research project is in collaboration with researchers from the University of Florida and the National Eye Institute. Ramamurthy and Ku aim to use their pre-clinical model to help treat childhood blindness known as leber congenital amaurosis (LCA).
“Our studies show that the severe blindness caused by rapid death of photoreceptor cells can be treated successfully with improved viral gene delivery systems. Gene replacement therapy for a similar disease is currently in phase II clinical trials for humans,” Ramamurthy said.
Lee Wiley, M.D., interim chair of the Eye Institute said, “This recent discovery by Dr. Ramamurthy’s vision research team provides new hope for vision restoration for those with LCA and will be a catalyst to finding better ways of treating other eye diseases.”
Ramamurthy’s research team continues to explore this treatment for patients who have a similar blinding disease but caused by different genetic mutations. This breakthrough in gene therapy will benefit treatment of other eye diseases as well.
Funding for the WVU Eye Institute Vision Research Center is provided in part by WV Lions Sight Conservation Foundation, Lions Clubs International Foundation, Research to Prevent Blindness, the National Institutes of Health, and from individual gifts to support our efforts to find the causes, treatment and prevention of eye diseases.
For more information about clinical trial opportunities, contact the Eye Institute’s research coordinator at 304-598-6956. To learn more about the WVU Eye Institute’s Vision Research Center, contact 304-598-4843.