Gene Therapy for Blindness

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05/02/2008

May. 02, 2008

Gene Therapy for Blindness

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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.

Produced by Annette Heist, Senior Producer

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

May. 02, 2008