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

Overview

Replacement Information

Key Specifications Table

CAS #Empirical Formula
775304-57-9C₁₅H₉FN₂O₃

Products

Catalog NumberPackaging Qty/Pack
5309180001 Glass bottle 25 mg
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
Catalog Number GTIN
5309180001 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.