Powder: -20°C for 3 years | In solvent: -80°C for 1 year
Ataluren (PTC124) 是一种可口服的 CFTR-G542X 无义等位基因抑制剂。
规格 | 价格/CNY | 货期 | 数量 | |
---|---|---|---|---|
5 mg | ¥ 255 | 现货 | ||
10 mg | ¥ 413 | 现货 | ||
25 mg | ¥ 739 | 现货 | ||
50 mg | ¥ 1,230 | 现货 | ||
100 mg | ¥ 1,960 | 现货 | ||
200 mg | ¥ 3,490 | 现货 | ||
500 mg | ¥ 5,580 | 现货 | ||
1 mL * 10 mM (in DMSO) | ¥ 455 | 现货 |
产品描述 | Ataluren (PTC124) is a novel, orally administered drug that targets nonsense mutations. Ataluren is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in patients aged 5 years and older who are able to walk. |
体外活性 | 在表达人CFTR-G542X转基因的Cftr -/- 小鼠中,皮下或口服给予PTC124(?60 mg/kg),以剂量依赖性方式抑制G542X无义突变,导致人 (h)CFTR蛋白的表达和功能的明显恢复,对无义介导的mRNA降解(NMD)或mRNA稳定性的其他方面没有任何影响.60 mg/kg PTC124处理Cftr+/+小鼠,观察到正常肠道跨上皮cAMP刺激的短路电流恢复29%,与庆大霉素相比具有的明显优势. |
体内活性 | 0.01-3 μM PTC124促进含有LUC-190无义等位基因的HEK293细胞中所有三个无义密码子的剂量依赖性通读,UGA读数最高,其次是UAG,然后是UAA,但它不抑制多重近端无义密码子。17 μM PTC124与稳定细胞系报告实验测定一致,PTC124(17 μM)促进Duchenne型肌营养不良症(DMD)患者或表达肌营养不良蛋白酶无义等位基因的MDXMDX小鼠的原代肌细胞中肌养蛋白的显著生成。PTC124选择性促进核糖体提前终止密码子而非正常终止密码子的通读,即使浓度大大高于达到最大活性所需值。与仅在更高浓度下有活性的庆大霉素相比,PTC124是更有效的无义抑制剂,并且相对于对照显示4至15倍通读刺激。PTC124与庆大霉素相似,在无义密码子后的嘧啶处(尤其是胞嘧啶,C) 活性最高。 |
细胞实验 | PTC124 (Ataluren) is prepared in DMSO and stored, and then diluted with appropriate medium (DMSO 1%) before use[2]. Duplicate samples of HEK293 cells harbouring LUC-190 (UGA) are incubated in the presence of 5?μM PTC124 (treated) or 1% DMSO (untreated) for 20?h. The cells are collected, washed twice in phosphate buffered saline (PBS), resuspended in sample buffer (Bio-Rad) and shipped on dry ice to Kendrick Laboratories for two-dimensional electrophoretic analysis Isoelectric focusing (pH?3.5-10) is carried out in glass tubes for 20,000 V-hours. One?μg of a tropomyosin internal standard is added to each sample. Second dimension SDS slab gel electrophoresis is carried out for approximately 6?h at 25?mA per gel. After electrophoresis, gels are transferred to PVDF paper. Computerized analysis of spot mobility used Phoretix software[2]. |
别名 | PTC124 |
分子量 | 284.24 |
分子式 | C15H9FN2O3 |
CAS No. | 775304-57-9 |
Powder: -20°C for 3 years | In solvent: -80°C for 1 year
H2O: < 1 mg/mL (insoluble or slightly soluble)
Ethanol: < 1 mg/mL (insoluble or slightly soluble)
DMSO: 53 mg/mL (186.5 mM)
可选溶剂 | 浓度 体积 质量 | 1 mg | 5 mg | 10 mg | 25 mg |
DMSO | 1 mM | 3.5182 mL | 17.5908 mL | 35.1815 mL | 87.9538 mL |
5 mM | 0.7036 mL | 3.5182 mL | 7.0363 mL | 17.5908 mL | |
10 mM | 0.3518 mL | 1.7591 mL | 3.5182 mL | 8.7954 mL | |
20 mM | 0.1759 mL | 0.8795 mL | 1.7591 mL | 4.3977 mL | |
50 mM | 0.0704 mL | 0.3518 mL | 0.7036 mL | 1.7591 mL | |
100 mM | 0.0352 mL | 0.1759 mL | 0.3518 mL | 0.8795 mL |
对于不同动物的给药剂量换算,您也可以参考 更多...
请在以下方框中输入您的动物实验信息后点击计算,可以得到母液配置方法和体内配方的制备方法: 比如您的给药剂量是10 mg/kg,每只动物体重20 g,给药体积100 μL,一共给药动物10 只,您使用的配方为5% DMSO+30% PEG300+5% Tween 80+60% ddH2O。那么您的工作液浓度为2 mg/mL。
母液配置方法:2 mg 药物溶于 50 μL DMSO (母液浓度为 40 mg/mL), 如您需要配置的浓度超过该产品的溶解度,请先与我们联系。
体内配方的制备方法:取 50 μL DMSO 主液,加入 300 μL PEG300, 混匀澄清,再加 50 μL Tween 80,混匀澄清,再加 600 μL ddH2O, 混匀澄清。
您可能有的问题的答案可以在抑制剂处理说明中找到,包括如何准备库存溶液,如何存储产品,以及基于细胞的分析和动物实验需要特别注意的问题。
Ataluren 775304-57-9 Autophagy Membrane transporter/Ion channel CFTR PTC124 PTC-124 PTC 124 inhibit Inhibitor Cystic fibrosis transmembrane conductance regulator inhibitor