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阿塔鲁伦 /Ataluren {[allProObj[0].p_purity_real_show]}

货号:A306827 同义名: PTC124

PTC-124 is nonsense allele inhibitor, used to treat Duchenne muscular dystrophy.

Ataluren 化学结构 CAS号:775304-57-9
Ataluren 化学结构
CAS号:775304-57-9
Ataluren 3D分子结构
CAS号:775304-57-9
Ataluren 化学结构 CAS号:775304-57-9
Ataluren 3D分子结构 CAS号:775304-57-9
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Ataluren 纯度/质量文件 产品仅供科研

货号:A306827 标准纯度: {[allProObj[0].p_purity_real_show]}
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产品名称 CFTR 其他靶点 纯度
Ataluren 98%
Lumacaftor ++++

F508del-CFTR, EC50: 0.1 μM

98%
CFTR(inh)-172 +++

CFTR, Ki: 300 nM

99%+
GlyH-101 +

CFTR, Ki: 4.3 μM

99%+
IOWH-032 ++

CFTR, IC50: 1.01 μM

99%+
Tezacaftor 99%+
1. 鼠标悬停在“+”上可以显示相关IC50的具体数值。"+"越多,抑制作用越强。2. "✔"表示该化合物对相应的亚型有抑制作用,但抑制强度暂时没有相关数据。

Ataluren 生物活性

靶点
  • CFTR

描述 Nonsense mutations have been implicated in numerous inherited diseases and several cancers. For example, cystic fibrosis (CF) is a common autosomal recessive disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. PTC-124 is an orally bioavailable compound that promotes readthrough of premature translation termination codons, suggesting its potential to treat genetic diseases caused by nonsense mutations like mutations in CFTR[3]. The minimal concentration of PTC-124 showing discernable readthrough was 0.01–0.1 μM (2.8-28 ng/ml). And it promoted dose-dependent readthrough of all three nonsense codons in stable cell lines harbouring LUC-190 nonsense alleles[4]. In a mouse model with CF, s.c. injection or oral administration of PTC-124 (60 mg/kg) to Cftr-/- mice expressing a human CFTR-G542X transgene suppressed the G542X nonsense mutation and restored a significant amount of human (h)CFTR protein and function. In addition, PTC-124 (60 mg/kg by s.c. injection or a liquid diet containing 0.9 mg/ml PTC-124) treatment restored 24-29% of the average cAMP-stimulated transepithelial chloride currents observed in wild-type mice. These results indicate that PTC-124 can effectively suppress the hCFTR-G542X nonsense mutation in vivo[3].

Ataluren 细胞研究

细胞系 浓度 检测类型 检测时间 活动说明 数据源
BAC_MMA∗EGFP 20 μM Function Assay 72 h  results in a significant increase in median peak fluorescence 23041189
HEK293T 10 µg/ml Function Assay restores full-length harmonin a1 (∼80 kDa) in p.R31X-transfected cells 23027640
ML1 3.3/10 μM Function Assay 48 h increases ARSB activity 22971959
ML2 3.3/10 μM Function Assay 48 h increases ARSB activity 22971959

Ataluren 临床研究

NCT号 适应症或疾病 临床期 招募状态 预计完成时间 地点
NCT03256799 Cystic Fibrosis Phase 4 Completed - United States, Alabama ... 展开 >> University of Alabama at Birmingham Birmingham, Alabama, United States, 35233 收起 <<
NCT02139306 Cystic Fibrosis Phase 3 Unknown November 2016 -
NCT01182324 - Completed - United States, Maryland ... 展开 >> National Human Genome Research Institute (NHGRI), 9000 Rockville Pike Bethesda, Maryland, United States, 20892 收起 <<

Ataluren 参考文献

[1]105(6):2064-9

[2]447(7140):87-91

Ataluren 实验方案

计算器
存储液制备 1mg 5mg 10mg

1 mM

5 mM

10 mM

3.52mL

0.70mL

0.35mL

17.59mL

3.52mL

1.76mL

35.18mL

7.04mL

3.52mL

Ataluren 技术信息

CAS号775304-57-9
分子式C15H9FN2O3
分子量 284.242
别名 PTC124
运输蓝冰
存储条件

液体 -20°C:3-6个月-80°C:12个月

粉末 Keep in dark place,Inert atmosphere,Room temperature

溶解度

DMSO: 50 mg/mL(175.91 mM),注意:DMSO长时间开封后,会吸水并导致溶解能力下降,请避免使用长期开封的DMSO

动物实验配方

IP 4% DMSO+2% Tween80+40% PEG300+water 1 mg/mL clear

PO 0.5% CMC-Na 22 mg/mL suspension

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