title Gene Repair Pill Update
description “In an advance online publication in the journal Nature, researchers describe developing a drug designed to be taken orally to treat diseases caused by “nonsense mutations,” which cause cells to prematurely stop making a needed protein. The drug, PTC124 is now in early human trials for treatment of nonsense mutations in Duchenne muscular dystrophy and cystic fibrosis. … researchers at PTC Therapeutics screened 800,000 small molecules to find a compound that tells cells to bypass the premature stop signs, while still obeying normal ones.” Video produced in 2005, but link features an interview about the new research.
producer Joyce Gramza for ScienCentral Video News
featuring JP Clancy, University of Alabama at Birmingham, cystic fibrosis patient Amanda Gosa
format Quicktime or RealVideo