Gene therapy may improve vision in congenital blindness LCA
1 shot of gene therapy and children with congenital blindness can now see — Leber’s congenital amaurosis (LCA) – Researchers from the University of Pennsylvania School of Medicine and the Center for Cellular and Molecular Therapeutics at the Children’s Hospital of Philadelphia have used gene therapy to safely improve vision in five children and seven adults with a rare form of congenital blindness.