Länk - Genreparerande tablett

published Apr 25, 2007 08:47 by admin ( last modified Apr 25, 2007 08:47 )

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.

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