Gene Therapy and Blindness

Listen

10/30/2009

Oct. 30, 2009

Gene Therapy and Blindness

Watch the Video - Navigating Obstacle Course »

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.

Produced by Flora Lichtman, Correspondent and Managing Editor, Video

Guests

Katherine A. High
Professor, Pediatrics

University of Pennsylvania School of Medicine

Director, Center for Cellular and Molecular Therapeutics

Children's Hospital of Philadelphia

Investigator, Howard Hughes Medical Institute

Philadelphia, Pennsylvania

Oct. 30, 2009