Molecular structure of PTC124, CAS Number: 775304-57-9
PTC124 (CAS 775304-57-9)
See product citations (2)
Alternate Names:
Ataluren
Application:
PTC124 is a firefly luciferase inhibitor and an inducer of ribosomal read-through of mRNAs
CAS Number:
775304-57-9
Purity:
≥99%
Molecular Weight:
284.20
Molecular Formula:
C15H9FN2O3
For Research Use Only. Not Intended for Diagnostic or Therapeutic Use.
* Refer to Certificate of Analysis for lot specific data.
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PTC124 may act to selectively induce ribosomal read-through of premature termination but not normal termination codons during mRNA translation. This compound has been reported to promote dystrophin production in primary muscle cells. Additionally, PTC124 may act to induce cAMP-stimulated transepithelial chloride currents in the murine apical surface of intestinal glands. Other experiments suggest that this agent is a potent inhibitor of firefly luciferase (FLuc).
PTC124 (CAS 775304-57-9) References
- PTC124 targets genetic disorders caused by nonsense mutations. | Welch, EM., et al. 2007. Nature. 447: 87-91. PMID: 17450125
- PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model. | Du, M., et al. 2008. Proc Natl Acad Sci U S A. 105: 2064-9. PMID: 18272502
- Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial. | Kerem, E., et al. 2008. Lancet. 372: 719-27. PMID: 18722008
- Mechanism of PTC124 activity in cell-based luciferase assays of nonsense codon suppression. | Auld, DS., et al. 2009. Proc Natl Acad Sci U S A. 106: 3585-90. PMID: 19208811
- Molecular basis for the high-affinity binding and stabilization of firefly luciferase by PTC124. | Auld, DS., et al. 2010. Proc Natl Acad Sci U S A. 107: 4878-83. PMID: 20194791
- Read-through strategies for suppression of nonsense mutations in Duchenne/ Becker muscular dystrophy: aminoglycosides and ataluren (PTC124). | Finkel, RS. 2010. J Child Neurol. 25: 1158-64. PMID: 20519671
- Ataluren (PTC124) induces cystic fibrosis transmembrane conductance regulator protein expression and activity in children with nonsense mutation cystic fibrosis. | Sermet-Gaudelus, I., et al. 2010. Am J Respir Crit Care Med. 182: 1262-72. PMID: 20622033
- Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis. | Wilschanski, M., et al. 2011. Eur Respir J. 38: 59-69. PMID: 21233271
- PTC124-mediated translational readthrough of a nonsense mutation causing Usher syndrome type 1C. | Goldmann, T., et al. 2011. Hum Gene Ther. 22: 537-47. PMID: 21235327
- A comparative evaluation of NB30, NB54 and PTC124 in translational read-through efficacy for treatment of an USH1C nonsense mutation. | Goldmann, T., et al. 2012. EMBO Mol Med. 4: 1186-99. PMID: 23027640
- A lack of premature termination codon read-through efficacy of PTC124 (Ataluren) in a diverse array of reporter assays. | McElroy, SP., et al. 2013. PLoS Biol. 11: e1001593. PMID: 23824517
- Toward a rationale for the PTC124 (Ataluren) promoted readthrough of premature stop codons: a computational approach and GFP-reporter cell-based assay. | Lentini, L., et al. 2014. Mol Pharm. 11: 653-64. PMID: 24483936
- Functional rescue of REP1 following treatment with PTC124 and novel derivative PTC-414 in human choroideremia fibroblasts and the nonsense-mediated zebrafish model. | Moosajee, M., et al. 2016. Hum Mol Genet. 25: 3416-3431. PMID: 27329764
Inhibitor of:
γ-sarcoglycan, 3β-HSD5, C19orf61, and Vmn2r72.Activator of:
γ2-Syntrophin, BBS7, CFTR, dystrophin, FRG1B, and utrophin.Ordering Information
| Product Name | Catalog # | UNIT | Price | Qty | FAVORITES | |
PTC124, 2 mg | sc-203222 | 2 mg | $203.00 | |||
PTC124, 5 mg | sc-203222A | 5 mg | $416.00 |