Friday, October 30th, 2009

Gene Therapy and Blindness

Watch an 8-year-old patient navigate an obstacle course 3 months after injection of the left eye. In the first clip, he uses his untreated eye (the other eye is patched). In the second clip, he uses his treated eye. (Credits: Courtesy of The Lancet. ) See More Videos

With just one injection of an experimental gene therapy treatment, doctors have been able to significantly improve the sight of 12 individuals born with a rare congenital vision disorder called Leber's congenital amaurosis. Several of the patients have improved so much that they are no longer classified as legally blind -- and one boy is now able to ride a bike and play baseball. The gene therapy advance is reported this week in an article in the journal The Lancet. We'll talk with one of the authors about the project, and about why this treatment seems to be so effective in children, while other gene therapy projects have struggled.