出典:Wikipedia
出典:『Wikipedia』 (2011/08/12 04:12 UTC 版)
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'.