Study shows gene therapy can restore vision to children with Leber congenital amaurosis

Results from a breakthrough study, conducted by researchers at The Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania School of Medicine, show that gene therapy has restored significant vision in five children and seven adults who were previously blind. The Foundation Fighting Blindness funded the clinical trial and has been the long standing funding source of the research that made the trial possible. The study used gene therapy to treat participants with Leber’s congenital amaurosis, a severe form of retinitis pigmentosa that causes blindness or substantial vision loss at birth. Trial participants demonstrated improvements in visual acuity, peripheral vision and light sensitivity. The greatest improvements occurred in four children ages 8-11, all of whom are now able to navigate an obstacle course in dim lighting. These findings have been reported in an online article in The Lancet, one of the world’s leading medical journals.

“These results are outstanding. We are delighted by not only the restoration of vision and the vision improvement that has been sustained, but also that the treatment has demonstrated an outstanding safety profile,” says Stephen Rose, Ph.D., chief research officer, Foundation Fighting Blindness.