5.30918

PTC-124

• 同義詞: PTC-124, 3-(5-(2-Fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoic acid, Premature Termination Codon Mutations Inhibitor, PTC124, Ataluren
• 經驗公式（希爾表示法）: C₁₅H₉FN₂O₃
• CAS號碼: 775304-57-9
• 分子量：: 284.24
• MDL號碼: MFCD09864996
• 分類程式碼代碼: 12352200
• NACRES: NA.77

屬性

• 化驗: ≥95% (HPLC)
• 品質等級: 100
• 形狀: solid
• 製造商／商標名: Calbiochem^®
• 儲存條件: OK to freeze; protect from light
• 顏色: white
• 溶解度: DMSO: 50 mg/mL
• 儲存溫度: 2-8°C
• SMILES 字串: Fc1c(cccc1)c2nc(n[o]2)c3cc(ccc3)C(=O)O
• InChI: 1S/C15H9FN2O3/c16-12-7-2-1-6-11(12)14-17-13(18-21-14)9-4-3-5-10(8-9)15(19)20/h1-8H,(H,19,20)
• InChI 密鑰: OOUGLTULBSNHNF-UHFFFAOYSA-N

描述

一般說明

A cell-permeable, non-toxic, orally bioavailable oxadiazole compound that is reported to promote read-through of premature, but not normal, translation termination codons and thereby suppress premature termination in protein synthesis caused by nonsense mutations (in ~10 to 100 nM with maximal activity reported at 3 µM). Promotes the suppression of nonsense alleles of the dystrophin gene in primary human muscle cells and in a mouse model of Duchenne muscular dystrophy (mdx). Also reported to rescue striated muscle function in mdx mice with a noticeable reduction in their serum creatine kinase values. Subcutaneous or oral administration of PTC124 to a mice expressing human CFTR-G542X transgene is shown to suppress G542X nonsense mutation and restore hCFTR function and cAMP-stimulated chloride channel activity (60 mg/kg for 3 weeks). Also acts as a potent and reversible inhibitor of purified firefly luciferase (FLuc; IC₅₀ ~ 10 nM) and protects it from tryptic degradation, but does not affect Renilla reniformis luciferase (RLuc).

生化／生理作用

Cell permeable: yes

Primary Target
PTC

Secondary Target
firefly luciferase

警告

Toxicity: Standard Handling (A)

重構

Following reconstitution, aliquot and freeze (-20°C). Stock solutions are stable for up to 6 months at -20°C.

其他說明

McElroy, S.P., et al. 2013. PLoS Biol.11, e1001593.
Auld, D.S., et al. 2010. Proc. Natl. Acad. Sci. USA.107, 4878.
Auld, D.S., et al. 2009. Proc. Natl. Acad. Sci. USA.106, 3585.
Du, M., et al. 2008. Proc. Natl. Acad. Sci. USA.105, 2064.

Welch, E.M., et al. 2007. Nature.447, 87.

法律資訊

CALBIOCHEM is a registered trademark of Merck KGaA, Darmstadt, Germany

安全資訊

• 象形圖: GHS07,GHS08
• 訊號詞: Warning
• 危險聲明: H336 - H373
• 防範說明: P260 - P271 - P304 + P340 + P312 - P314 - P403 + P233 - P405
• 危險分類: STOT RE 2 - STOT SE 3
• 標靶器官: Central nervous system, Kidney
• 儲存類別代碼: 11 - Combustible Solids
• 水污染物質分類（WGK）: WGK 3
• 閃點（°F）: Not applicable
• 閃點（°C）: Not applicable