5.30918

PTC-124

• Sinónimos: PTC-124, 3-(5-(2-Fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoic acid, Premature Termination Codon Mutations Inhibitor, PTC124, Ataluren
• Fórmula empírica (notación de Hill): C₁₅H₉FN₂O₃
• Número de CAS: 775304-57-9
• Peso molecular: 284.24
• Código UNSPSC: 12352200
• NACRES: NA.77

Propiedades

• Análisis: ≥95% (HPLC)
• Nivel de calidad: 100
• formulario: solid
• fabricante / nombre comercial: Calbiochem^®
• condiciones de almacenamiento: OK to freeze; protect from light
• color: white
• solubilidad: DMSO: 50 mg/mL
• temp. de almacenamiento: 2-8°C
• 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)
• Clave InChI: OOUGLTULBSNHNF-UHFFFAOYSA-N

Descripción

Descripción general

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).

Acciones bioquímicas o fisiológicas

Cell permeable: yes

Primary Target
PTC

Secondary Target
firefly luciferase

Advertencia

Toxicity: Standard Handling (A)

Reconstitución

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

Otras notas

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.

Información legal

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

Información de seguridad

• Pictogramas: GHS07,GHS08
• Palabra de señalización: Warning
• Frases de peligro: H336 - H373
• Consejos de prudencia: P260 - P271 - P304 + P340 + P312 - P314 - P403 + P233 - P405
• Clasificaciones de peligro: STOT RE 2 - STOT SE 3
• Órganos de actuación: Central nervous system, Kidney
• Código de clase de almacenamiento: 11 - Combustible Solids
• Clase de riesgo para el agua (WGK): WGK 3
• Punto de inflamabilidad (°F): Not applicable
• Punto de inflamabilidad (°C): Not applicable