5.30918
PTC-124
别名:
PTC-124, 3-(5-(2-Fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoic acid, Premature Termination Codon Mutations Inhibitor, PTC124, Ataluren
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选择规格
变更视图
关于此项目
经验公式（希尔记法）:
C15H9FN2O3
化学文摘社编号:
分子量:
284.24
MDL number:
UNSPSC Code:
12352200
NACRES:
NA.77
Assay:
≥95% (HPLC)
Form:
solid
Storage condition:
OK to freeze, protect from light
assay
≥95% (HPLC)
Quality Segment
form
solid
manufacturer/tradename
Calbiochem®
storage condition
OK to freeze, protect from light
color
white
solubility
DMSO: 50 mg/mL
storage temp.
2-8°C
SMILES string
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 key
OOUGLTULBSNHNF-UHFFFAOYSA-N
General description
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).
Biochem/physiol Actions
Cell permeable: yes
Primary Target
PTC
PTC
Secondary Target
firefly luciferase
firefly luciferase
Preparation Note
Following reconstitution, aliquot and freeze (-20°C). Stock solutions are stable for up to 6 months at -20°C.
Other Notes
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.
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.
Legal Information
CALBIOCHEM is a registered trademark of Merck KGaA, Darmstadt, Germany
Disclaimer
Toxicity: Standard Handling (A)
警示词
Warning
Hazard Classifications
STOT RE 2 - STOT SE 3
靶器官
Central nervous system, Kidney
存储类别
11 - Combustible Solids
怎么回事？
WGK 3
闪点 (F)
Not applicable
闪点 (°C)
Not applicable
