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530918 PTC-124 - CAS 775304-57-9 - Calbiochem

PTC-124 - CAS 775304-57-9 - Calbiochem MSDS (material safety data sheet) or SDS, CoA and CoQ, dossiers, brochures and other available documents.

Synonyms: Ataluren, 3-(5-(2-Fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoic acid, Premature Termination Codon Mutations Inhibitor, PTC124

Primary Target: PTC
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530918
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Overview

Replacement Information

Key Spec Table

CAS #Empirical Formula
775304-57-9C₁₅H₉FN₂O₃

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      Description
      OverviewA 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).
      Catalogue Number530918
      Brand Family Calbiochem®
      SynonymsAtaluren, 3-(5-(2-Fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoic acid, Premature Termination Codon Mutations Inhibitor, PTC124
      References
      ReferencesMcElroy, 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.
      Product Information
      CAS number775304-57-9
      FormWhite solid
      Hill FormulaC₁₅H₉FN₂O₃
      Chemical formulaC₁₅H₉FN₂O₃
      ReversibleY
      Quality LevelMQ100
      Applications
      Biological Information
      Primary TargetPTC
      Secondary targetfirefly luciferase
      Purity≥95% by HPLC
      Physicochemical Information
      Cell permeableY
      Dimensions
      Materials Information
      Toxicological Information
      Safety Information according to GHS
      Safety Information
      Product Usage Statements
      Storage and Shipping Information
      Ship Code Ambient Temperature Only
      Toxicity Standard Handling
      Storage +2°C to +8°C
      Protect from Light Protect from light
      Do not freeze Ok to freeze
      Special InstructionsFollowing reconstitution, aliquot and freeze (-20°C). Stock solutions are stable for up to 6 months at -20°C.
      Packaging Information
      Packaged under inert gas Packaged under inert gas
      Transport Information
      Supplemental Information
      Specifications
      Global Trade Item Number
      Catalogue Number GTIN
      5.30918.0001 04055977283242

      Documentation

      PTC-124 - CAS 775304-57-9 - Calbiochem SDS

      Title

      Safety Data Sheet (SDS) 

      References

      Reference overview
      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.

      Technical Info

      Title
      White Paper: Further considerations of antibody validation and usage.
      Data Sheet

      Note that this data sheet is not lot-specific and is representative of the current specifications for this product. Please consult the vial label and the certificate of analysis for information on specific lots. Also note that shipping conditions may differ from storage conditions.

      Revision05-January-2015 JSW
      SynonymsAtaluren, 3-(5-(2-Fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoic acid, Premature Termination Codon Mutations Inhibitor, PTC124
      DescriptionA 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).
      FormWhite solid
      Intert gas (Yes/No) Packaged under inert gas
      CAS number775304-57-9
      Chemical formulaC₁₅H₉FN₂O₃
      Purity≥95% by HPLC
      SolubilityDMSO (50 mg/ml)
      Storage Protect from light
      +2°C to +8°C
      Do Not Freeze Ok to freeze
      Special InstructionsFollowing reconstitution, aliquot and freeze (-20°C). Stock solutions are stable for up to 6 months at -20°C.
      Toxicity Standard Handling
      ReferencesMcElroy, 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.