Leber's Congenital Amaurosis

Meet two of the scientists behind the game-changing gene therapies created by the Research Institute's Clinical Vector Core.

A groundbreaking clinical trial of gene therapy for a form of congenital blindness, sponsored by The Children’s Hospital of Philadelphia in collaboration with Penn Medicine, was recently recognized with the Distinguished Clinical Research Achievement Award from the Clinical Research Forum, an organization of clinical research centers, industry, and volunteer groups.