Gene Therapy for Blindness (broadcast Friday, May 2nd, 2008)

A retinal fundus photograph of a Briard dog with congenital stationary night blindness. Mutations to the same gene in humans, RPE65, cause Leber congenital amaurosis. G. Aguirre/Cornell University. Copyright (c) Cornell University

Researchers report some success in using a gene therapy technique to treat one form of congenital blindness. Two teams of scientists have used modified viruses injected into the eye to partially restore vision to people suffering from Leber congenital amaurosis, or LCA, a congenital illness that results in the degradation of the retina. People with LCA slowly lose all vision, and typically are completely blind by their 20s or 30s. The modified virus used in the therapy carries a replacement gene to the retina, allowing it to produce a missing protein responsible for the disease. In this segment, Ira talks with one of the authors of one report on the research, which was published this week in two studies in the New England Journal of Medicine. Teachers, find more information about using Science Friday as a classroom resource in the Kids' Connection.