Gene Repair Pill Update

04/23/2007

ScienCentral News
April 23, 2007


In an advance online publication in the journal Nature, researchers 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, PTC-124 is now in early human trials for treatment of nonsense mutations in Duchenne Muscular Dystrophy and Cystic Fibrosis.

The paper describes how 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.

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