The FDA panel approved new gene therapy Luxturna. The treatment for Leber congenital amaurosis (LCA) an inherited retinal disease caused by defects in a gene called RPE65. Parents can unknowingly carry the flawed gene for generations.
Patients with a rare inherited form of blindness could be able to regain their sight if the Food and Drug Administration approves an experimental gene therapy in January.
https://spinonews.com/2017/12/20/fda-granted-gene-therapy-luxturna-for-vision-loss-and-blindness/