PTC124
Jump to navigation
Jump to search
Indications
- genetic diseases caused by nonsense mutations resulting in premature termination codon
Dosage
oral
Adverse effects
- none reported
Mechanism of action
- prevents premature termination of mRNA translation due to premature termination codon resulting from nonsense mutation
- does NOT interfere with true stop codon
More general terms
References
- ↑ Welch EM et al, PTC124 targets genetic disorders caused by nonsense mutations Nature 2007, 447:87 PMID: https://www.ncbi.nlm.nih.gov/pubmed/17450125
Schmitz A and Famulok M Chemical biology: Ignore the nonsense. Nature 2007, 447:42 PMID: https://www.ncbi.nlm.nih.gov/pubmed/17450128