Ataluren, formerly known as PTC124, is a novel small-molecular agent designed to make ribosomes become less sensitive to, or possibly ignore premature stop codons.
This may be particularly beneficial in genetic disorders where the mRNA contains a mutation causing premature stop codon or nonsense codon. However,
it is not equally effective with every stop codon, working best on the sequence 'UGA' 1. PTC124 has been tested on healthy humans and humans carrying
genetic disorders caused by nonsense mutations1,2, such as some people with cystic fibrosis and Duchenne muscular dystrophy
1. Welch EM, Barton ER, Zhuo J et al. PTC124 targets genetic disorders caused by nonsense mutations. Nature 447 (7140): 87–91
2.
Hirawat S, Welch EM, Elfring GL et al. Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers". Journal of clinical pharmacology 47 (4): 430–444
Product Specification
M.Wt: 284.24 Formula: C15H9FN2O3 Solubility: DMSO Purity: >99% Storage: at -20℃ 2 years CAS No.: 775304-57-9
|