compound introduction
Ataluren (PTC124)选择性诱导核糖体通读，不影响正常的终止密码子，EC50为0.1 μM，可以治疗nonsense mutations（无义突变） (如CFTR无义突变引起的囊性纤维病(CF))造成的遗传性疾病。A firefly luciferase inhibitor and an inducer of ribosomal read-throμgh of mRNAs. PTC124 (Ataluren) selectively induces ribosomal read-through of premature but not normal termination codons, with EC50 of 0.1 μM, may provide treatment for genetic disorders caused by nonsense mutations (e.g. CF caused by CFTR nonsense mutation). A firefly luciferase inhibitor and an inducer of ribosomal read-through of mRNAs.
