| 应用 |
A firefly luciferase inhibitor and an inducer of ribosomal read-through of mRNAs. |
| 产品介绍 |
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。 |
| 备注 |
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 mutat |
| 生化机理 |
PTC124 has been shown to selectively induce ribosomal read-through of premature termination but not normal termination codons during mRNA translation. This compound has been reported to promote dystrophin production in primary muscle cells. Additionally, PTC124 has been shown to induce cAMP-stimulated transepithelial chloride currents in the murine apical surface of intestinal glands. Other experiments suggest that this agent is a potent inhibitor of firefly luciferase (FLuc). |
| 别名 |
阿塔鲁伦;3-[5-(2-氟苯基)-1,2,4-恶二唑-3-基]苯甲酸;Ataluren; PTC-124; PTC 124;3-[5-(2-Fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid |
