| 货号 | 16758-5mg |
| 描述 | Nonsense mutations create a premature termination of mRNA translation and have been implicated in various genetic disorders, including muscular dystrophy and cystic fibrosis.1 PTC-124 is a nonaminoglycoside that has been reported to selectively induce ribosomes to read through premature nonsense stop signals on mRNA, thus allowing the production of full-length, functional proteins.1 In a mouse model of cystic fibrosis caused by nonsense mutations, PTC-124 treatment (60 mg/kg s.c. injection or 0.3-0.9 mg/ml orally) has been shown to restore cystic fibrosis transmembrane conductance regulator (CFTR) protein expression and function.2 The target activity of PTC-124 was initially evaluated by firefly luciferase reporter cell-based nonsense codon assay (IC50 = 7 nM); however, subsequent assessments using a Renilla reniformis luciferase reporter have failed to produce nonsense codon suppression activity.3 Thus, while PTC-124 is in clinical testing in patients with nonsense mutations within the CFTR or dystrophin genes, controversy surrounds its exact mechanism of action.1,3,4,5 |
| 别名 | Ataluren;Translarna™; |
| 供应商 | Cayman |
| 应用文献 | |
| 1.Finkel, R.S. Read-through strategies for suppression of nonsense mutations in Duchenne/Becker muscular dystrophy: Aminoglycosides and ataluren (PTC124). J.Child Neurol. 25(9), 1158-1164 (2010). 2.Du, M.,Liu, X.,Welch, E.M., et al. PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model. Proceedings of the National Academy of Sciences of the United States of America 105(6), 2064-2069 (2008). 3.Auld, D.S.,Thorne, N.,Maguire, W.F., et al. Mechanism of PTC124 activity in cell-based luciferase assays of nonsense codon suppression. Proceedings of the National Academy of Sciences of the United States of America 106(9), 3585-3590 (2009). 4.Wilschanski, M.,Miller, L.L.,Shoseyov, D., et al. Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis. European Respiratory Journal 38(1), 59-69 (2011). 5.McElroy, S.P.,Nomura, T.,Torrie, L.S., et al. A lack of premature termination codon read-through efficacy of PTC124 (Ataluren) in a diverse array of reporter assays. PLOS Biology 11(6), 1-8 (2013). | |
| 运输条件 | Room temperature in continental US; may vary elsewhere |
| 存放说明 | -20 |
| 纯度 | ≥98% |
| 计算分子量 | 284.3 |
| 分子式 | C15H9FN2O3 |
| CAS号 | 775304-57-9 |
| 稳定性 | ≥ 2 years |
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