Gene therapy efficacy for LCA: Improvement is followed by decline in vision

(University of Pennsylvania School of Medicine) Gene therapy for Leber congenital amaurosis, an inherited disorder that causes loss of night- and day-vision starting in childhood, improved patients' eyesight within weeks of treatment in a clinical trial of 15 children and adults at the Scheie Eye Institute at the Perelman School of Medicine at the University of Pennsylvania. New results involving a subset of patients from the ongoing trial show these benefits peaked one to three years after trea...