Gene Therapy for Leber's Congenital Amaurosis

Gene therapy is when we use DNA, rather than a drug or surgery, to treat a condition. In the case of LCA2, a harmless virus is used to deliver a new, normal copy of the RPE65 gene to those cells which need it (i.e. the RPE cells) with the aim of helping the cells to gain more normal function.

Results from the world's first gene therapy trial for inherited blindness reveal that the eyesight of people with Leber congenital amaurosis (LCA) can be improved at least in the short term.