生物活性
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。与 Gentamicin只在较高浓度有活性相比，PTC124是更有效的无义抑制剂，与对照组相比，通读刺激强4-15倍。PTC124(0.01-3 μM)作用于 含LUC-190无义等位基因的HEK293细胞，促进三个无义密码子的通读，这种作用存在剂量依赖性，在UGA通读最高，然后是UAG，再然后是UAA,但是不会抑制多种近端无义密码子。与Gentamicin相似, PTC124在无义密码子后的嘧啶处(尤其是胞嘧啶,C) 活性最高。
化学数据
| 分子量 | 284.24 |
| 分子式 | C15H9FN2O3 |
| CAS号 | 775304-57-9 |
| 纯度 | >99% |
| 溶解性（25°C） | DMSO ≥47 mg/mL |
| 储存和运输条件 | 固体粉末： -20°C 冷藏长期储存
常温运输及临时存放 |
实验操作 来自于公开的文献，仅供相同实验参考（如实验材料、目的不同，请参考其他文献）
| 细胞实验 |
|---|
| 细胞系 | Grip-Tite 293 cells transfected with the pFLuc190UGA construct |
| 方法 | Luciferase Reporter Assays. All cell-based assays were performed in 1,536-well plates with an assay volume of 4.5 μL per well. Comparative cell-based experiments were performed with cells from the same batch of transfected cells to control for transfection efficiency. Cells were incubated with compound or antibiotic for 16–72 h. In experiments with Grip-Tite cells, cells were washed with PBS before the addition of detection reagent containing the FLuc or RLuc substrate. Luminescence from luciferase activity was detected by using ViewLux (PerkinElmer). Experimental plates contained the transcriptional activator MS-275 as a positive control and DMSO-treated cells or mock-transfected cells as a negative control. Percentage activity was defined as the percentage signal above DMSO-treated cells. |
| 浓度 | 0~100μM |
| 处理时间 | 24 hr |
| 动物实验 |
|---|
| 动物模型 | Cftr–/– hCFTR-G542X mice |
| 配制 | saline |
| 剂量 | 15, 30 or 60mg/kg initiated 16 days after birth and continued once daily until the animals were killed |
| 给药处理 | Subcutaneous injections |
不同实验动物依据体表面积的等效剂量转换表（数据来源于FDA指南）
| 小鼠 | 大鼠 | 兔 | 豚鼠 | 仓鼠 | 狗 |
| 重量 (kg) | 0.02 | 0.15 | 1.8 | 0.4 | 0.08 | 10 |
| 体表面积 (m2) | 0.007 | 0.025 | 0.15 | 0.05 | 0.02 | 0.5 |
| Km系数 | 3 | 6 | 12 | 8 | 5 | 20 |
| 动物 A (mg/kg) = 动物 B (mg/kg) × | 动物 B的Km系数 |
| 动物 A的Km系数 |
例如，依据体表面积折算法，将化合物用于小鼠的剂量20 mg/kg 换算成大鼠的剂量，需要将20 mg/kg 乘以小鼠的Km系数（3），再除以大鼠的Km系数（6），得到化合物用于大鼠的等效剂量为10 mg/kg。
储备液配制
以下数据基于产品分子量，对于特殊产品，请参照COA中的储备液配制条件和说明进行操作。
| Concentration / Solvent Volume / Mass | 1 mg | 5 mg | 10 mg |
|---|
| 1 mM | 3.5182 mL | 17.5908 mL | 35.1815 mL |
| 5 mM | 0.7036 mL | 3.5182 mL | 7.0363 mL |
| 10 mM | 0.3518 mL | 1.7591 mL | 3.5182 mL |
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