Phase 1 dose escalation study on gene therapy treatment for LCA shows importance of early treatment

A gene therapy treatment of 12 patients between the ages of 8-44 years suffering RPE65-associated Leber’s Congenital Amaurosis (LCA) has been shown to improve vision in all participants. The report noted that “patients had at least a 2 log unit increase in pupillary light responses and an 8 year old child had nearly the same level of light sensiti