PTC124

Indications: - genetic diseases caused by nonsense mutations resulting in premature termination codon a) cystic fibrosis ? b) muscular dystrophy ? Dosage: oral Adverse effects: - none reported Mechanism of action: 1) prevents premature termination of mRNA translation due to premature termination codon resulting from nonsense mutation 2) does NOT interfere with true stop codon

References

- Welch EM et al, PTC124 targets genetic disorders caused by nonsense mutations Nature 2007, 447:87 PMID: 17450125 - Schmitz A and Famulok M Chemical biology: Ignore the nonsense. Nature 2007, 447:42 PMID: 17450128