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WVU Eye Institute Researchers Restore Vision with Gene Replacement Therapy

 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.”

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