genetic therapy

On Tuesday, the U.S. Food and Drug Administration approved a breakthrough genetic therapy for a heredity condition that causes blindness. The treatment, Luxturna, was approved ahead of schedule, thanks, in part, to work done by researchers at the University of Iowa. Prior to the approval of Luxturna, there was no treatment for the rare condition, known as Leber congenital amaurosis (LCA). About 6,000 people have LCA worldwide, including 1,000 to 2,000 people in the United States.