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Every gene has a stop sign that signals the ribosome that the full peptide has been created and that no additional amino acids should be added. Some important diseases, like cystic fibrosis (CF) and muscular dystrophy, are caused by nonsense mutations in a single gene that create “premature stop signs.” As a result, insufficient amounts of an essential peptide are formed. Such stop signs are formally called premature termination codons, or PTCs.
A biotech company and multiple academic institutions have developed a novel molecule called PTC124 that causes the ribosome to ignore the PTC and to continue making the full peptide. Importantly, PTC124 does not interfere with the action of true stop signs or cause any apparent adverse effects. In bo…