PTC-124

• CAT Number: I004853
• CAS Number: 775304-57-9
• Molecular Formula: C₁₅H₉FN₂O₃
• Molecular Weight: 284.24
• Purity: ≥95%
• Target: CFTR
• Solubility: DMSO: ≥ 52 mg/mL
• Storage: 3 years -20C powder
• IUPAC Name: 3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid
• InChI: 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)
• InChIKey: OOUGLTULBSNHNF-UHFFFAOYSA-N
• SMILES: C1=CC=C(C(=C1)C2=NC(=NO2)C3=CC(=CC=C3)C(=O)O)F

PTC124 (Cat No.: I004853) selectively induces ribosomal transcriptional read-through but does not affect normal stop codons. The EC50 in HEK293 cells is 0.1 μM and can treat families caused by nonsense mutations (such as CF caused by CFTR nonsense mutations) genetic disease. If you need, please contact us in time, and we will reply to you as soon as possible within 24 hours.

Reference

1:Aminoglycosides, but not PTC124 (Ataluren), rescue nonsense mutations in the leptin receptor and in luciferase reporter genes. Bolze F, Mocek S, Zimmermann A, Klingenspor M.Sci Rep. 2017 Apr 21;7(1):1020. doi: 10.1038/s41598-017-01093-9. PMID: 28432296 Free PMC Article2:Readthrough-Promoting Drugs Gentamicin and PTC124 Fail to Rescue Nav1.5 Function of Human-Induced Pluripotent Stem Cell-Derived Cardiomyocytes Carrying Nonsense Mutations in the Sodium Channel Gene SCN5A. Kosmidis G, Veerman CC, Casini S, Verkerk AO, van de Pas S, Bellin M, Wilde AA, Mummery CL, Bezzina CR.Circ Arrhythm Electrophysiol. 2016 Nov;9(11). pii: e004227. PMID: 27784737 3: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, Tracey-White D, Smart M, Weetall M, Torriano S, Kalatzis V, da Cruz L, Coffey P, Webster AR, Welch E.Hum Mol Genet. 2016 Aug 15;25(16):3416-3431. doi: 10.1093/hmg/ddw184. Epub 2016 Jun 21. PMID: 27329764 4:Enhancement of premature stop codon readthrough in the CFTR gene by Ataluren (PTC124) derivatives. Pibiri I, Lentini L, Melfi R, Gallucci G, Pace A, Spinello A, Barone G, Di Leonardo A.Eur J Med Chem. 2015 Aug 28;101:236-44. doi: 10.1016/j.ejmech.2015.06.038. Epub 2015 Jun 21. PMID: 26142488 5:Therapeutic effect of prenatal alkalization and PTC124 in Na(+)/HCO3(-) cotransporter 1 p.W516* knock-in mice. Fang YW, Yang SS, Chau T, Nakamura M, Yamazaki O, Seki G, Yamada H, Hsu HM, Cheng CJ, Lin SH.Gene Ther. 2015 May;22(5):374-81. doi: 10.1038/gt.2015.7. Epub 2015 Feb 26. PMID: 25716530 6:Toward a rationale for the PTC124 (Ataluren) promoted readthrough of premature stop codons: a computational approach and GFP-reporter cell-based assay. Lentini L, Melfi R, Di Leonardo A, Spinello A, Barone G, Pace A, Palumbo Piccionello A, Pibiri I.Mol Pharm. 2014 Mar 3;11(3):653-64. doi: 10.1021/mp400230s. Epub 2014 Feb 7. PMID: 24483936 Free PMC Article7:Comparison of read-through effects of aminoglycosides and PTC124 on rescuing nonsense mutations of HERG gene associated with long QT syndrome. Yu H, Liu X, Huang J, Zhang Y, Hu R, Pu J.Int J Mol Med. 2014 Mar;33(3):729-35. doi: 10.3892/ijmm.2013.1601. Epub 2013 Dec 23. PMID: 24366185 8:A lack of premature termination codon read-through efficacy of PTC124 (Ataluren) in a diverse array of reporter assays. McElroy SP, Nomura T, Torrie LS, Warbrick E, Gartner U, Wood G, McLean WH.PLoS Biol. 2013;11(6):e1001593. doi: 10.1371/journal.pbio.1001593. Epub 2013 Jun 25. PMID: 23824517 Free PMC Article9:A comparative evaluation of NB30, NB54 and PTC124 in translational read-through efficacy for treatment of an USH1C nonsense mutation. Goldmann T, Overlack N, Möller F, Belakhov V, van Wyk M, Baasov T, Wolfrum U, Nagel-Wolfrum K.EMBO Mol Med. 2012 Nov;4(11):1186-99. doi: 10.1002/emmm.201201438. Epub 2012 Oct 2. PMID: 23027640 Free PMC Article10:The chemical compound PTC124 does not affect cellular electrophysiology of cardiac ventricular myocytes. Koopmann TT, Verkerk AO, Bezzina CR, de Bakker JM, Wilde AA.Cardiovasc Drugs Ther. 2012 Feb;26(1):41-5. doi: 10.1007/s10557-011-6352-2. PMID: 22065308