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An in vitro study of PTC124, a drug in clinical trials for the treatment of cystic fibrosis and several other genetic disorders, has some scientists wondering whether the molecule works the way its developers say it does. PTC124 is intended to treat disorders, including some cases of cystic fibrosis and Duchenne muscular dystrophy, that stem from mutations that cause stop codons to erroneously appear in critical gene transcripts, thereby inhibiting translation of vital proteins. The new study, published today (June 25) in PLOS Biology, demonstrates that PTC124 does not promote read-through of such premature stop codons, or “nonsense” mutations, in several reporter assays done using a human cell line. “The paper is well designed and carefully executed, and the dataset is unambiguous,” said Bryan Roth, a professor of pharmacology at the University of North Carolina at Chapel Hill who was not involved in the work, in an email to The Scientist.
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