H2617007计算溶液所需的质量、体积或浓度。
有货
概述
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.
Information
Ataluren (PTC124) Ataluren (PTC124) selectively induces ribosomal read-through of premature but not normal termination codons, with EC50 of 0.1 μM in HEK293 cells, may provide treatment for genetic disorders caused by nonsense mutations (e.g. CF caused
In vitro
Compared with Gentamicin which is only active at much higher concentrations, PTC124 is a more potent nonsense-suppressing agent and exhibits 4- to 15-fold stimulation of read-through relative to controls. PTC124 (0.01-3 μM) promotes dose-dependent read-through of all three nonsense codons in HEK293 cells harboring LUC-190 nonsense alleles with the highest read-through at UGA, followed by UAG and then UAA, but it does not suppress multiple proximal nonsense codons. Like Gentamicin, PTC124 is most active when a pyrimidine (in particular cytosine, C) follows the nonsense codon. Consistent with the stable cell line reporter assay, PTC124 (17 μM) promotes significant production of dystrophin in primary muscle cells from Duchenne muscular dystrophy (DMD) patients or MDXMDX mice expressing dystrophin nonsense alleles. PTC124 selectively promotes ribosomal read-through of premature termination but not normal termination codons, even at concentrations substantially greater than the values achieving maximal activity.
In vivo
Due to functional recovery of dystrophin production, oral, intraperitoneal or combined dosing of PTC124 for 2-8 weeks partially rescues functional strength deficit in dystrophic muscles of MDX mice, and results in partial protection against contraction-induced injury in the extensor digitorum longus (EDL) muscles, as well as significant reductions in serum creatine kinase values. In Cftr-/- mice expressing a human CFTR-G542X transgene, subcutaneous or oral administration of PTC124 (~60 mg/kg) suppresses the G542X nonsense mutation in a dose-dependent manner, leading to a significant restoration of human (h)CFTR protein expression and function without any effect on nonsense-mediated mRNA decay (NMD) or other aspects of mRNA stability. PTC124 treatment (60 mg/kg) restores 29% of the normal intestinal transepithelial cAMP-stimulated shortcircuit currents observed in Cftr+/+ mice, displaying a significant advantage compared with Gentamicin.
Cell Data
cell lines：
Concentrations：
Incubation Time：
Powder Purity:≥99%
规格
别名
阿塔鲁伦 (PTC124)
英文别名
3-(5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoic acid
规格或纯度
Moligand™, 10mM in DMSO
英文名称
Ataluren (PTC124)
生化机理
Ataluren (PTC124) 在 HEK293 细胞中的 EC50 为 0.1 μM，可选择性地诱导过早终止密码子而非正常终止密码子的核糖体通读，可用于治疗无义突变引起的遗传疾病（如 CFTR 无义突变引起的 CF）。第 3 阶段。
储存条件
-80℃储存
运输条件
特低温运输
作用类型
调节剂
作用机制
80S 核糖体调节器
名称和识别符
分子类型
小分子
Isomeric SMILES
C1=CC=C(C(=C1)C2=NC(=NO2)C3=CC(=CC=C3)C(=O)O)F
分子量
284.24
Reaxy-Rn
11000909
Reaxys-RN link address
https://www.reaxys.com/reaxys/secured/hopinto.do?context=S&query=IDE.XRN=11000909&ln=
高级数据
产品属性
| ALogP | 3.1 |
|---|
化学和物理性质
熔点
242°C
安全和危险性（GHS）
象形图
信号词
警告
危险声明
H227: 可燃液体
预防措施声明
P210: 远离热源，热表面，火花，明火和其他点火源。 - 禁止抽烟。
P280: 戴防护手套/穿防护服/戴防护眼罩/戴防护面具。
P370+P378: 火灾时：使用干砂、干粉或抗醇泡沫灭火。
P403+P235: 存放在通风良好的地方。保持低温。
P501: 将内容物/容器送到批准的废物处理厂处理
质检证书(CoA,COO,BSE/TSE 和分析图谱)
技术文档和文章
此产品的引用文献
溶液计算器
产品仅提供给经过验证的企业、机构和合格的专业人员用于研发。不适用于人类、动物、诊断或治疗。
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危险品化学品经营许可证（带存储）