- GlyH-101
- VX-661
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- Ivacaftor (VX-770)
| PTC124 (Ataluren)CFTR-G542X nonsense allele inhibitor |
Sample solution is provided at 25 µL, 10mM.
Nature.2017 Jan 19;541(7637):417-420.
Nature.2018 Nov;563(7731):407-411.Nature.2018 Jun 13.Nature.2018 Jun 27.Nature.2018 Mar 29;555(7698):673-677.Nature.2017 Sep 7;549(7670):96-100.Nature.2016 Apr 21;532(7599):398-401.
Science.2016 Aug 5;353(6299)594-8Nat Nanotechnol.2017 Dec;12(12):1190-1198.Nature Biotechnology.2017 Jun;35(6):569-576Nat Med.2018 Sep 17.Cell.2018 Dec 21. pii: S0092-8674(18)31561-7.Cell.Available online 25 October 2018.Cell.2018 Sep 27. pii: S0092-8674(18)31183-8.Cell.2018 Jun 28;174(1):172-186.e21.Cell.2018 Feb 22;172(5):1007-1021.e17.Cell.2017 Nov 30;171(6):1284-1300.e21.Cell.2017 Aug 17. pii: S0092-8674(17)30869-3.Cell.2017 Jul 13;170(2):312-323Nat Med.2018 Jan 29.Nat Med.2017 Nov;23(11):1342-1351.Cell.2017 Apr 6;169(2):286-300.Cell.2015 Aug 27;162(5):987-1002.Cell.2015 Feb 12;160(4):729-44.Nature Medicine.2017 Apr;23(4):493-500.Cancer Cell.2018 May 14;33(5):905-921.e5.Cancer Cell.2018 Apr 9;33(4):752-769.e8.Cancer Cell.2018 Mar 12;33(3):401-416.e8.Cancer Cell.2017 Aug 14;32(2):253-267.e5.Nat Methods.2018 Jul;15(7):523-526.Cell Stem Cell.2018 May 3;22(5):769-778.e4.Cell Stem Cell.2017 Nov 20. pii: S1934-5909(17)30375-2.Quality Control & MSDS
- View current batch:
- Purity = 98.00%
- COA (Certificate Of Analysis)
- MSDS (Material Safety Data Sheet)
- Datasheet
Chemical structure
| Description | PTC124 (Ataluren) is a selective inducer of ribosomal read-through of premature with an EC50 value of 0.1 μM. | |||||
| Targets | CFTR | |||||
| IC50 | ||||||
| Cell experiment: [1] | |
Cell lines | HEK293 cells |
Preparation method | The solubility of this compound in DMSO is >10 mM. General tips for obtaining a higher concentration: Please warm the tube at 37 °C for 10 minutes and/or shake it in the ultrasonic bath for a while.Stock solution can be stored below -20°C for several months. |
Reaction Conditions | 3 μM, 16 hours |
Applications | Cultured HEK293 cells harbouring UAA, UAG or UGA LUC-190 nonsense alleles were treated with increasing concentrations of PTC124 for 16 h, and assayed for luciferase activity. PTC124 promoted dose-dependent readthrough of all three nonsense codons. Levels of suppression correlated inversely with established termination efficiencies, with the highest readthrough at UGA, followed by UAG and then UAA. The minimal concentration of PTC124 showing discernable readthrough was 0.01–0.1 μM, whereas the concentration promoting maximal activity was approximately 3 μM. |
| Animal experiment: [2] | |
Animal models | Cftr-/- hCFTR-G542X Mice |
Dosage form | Subcutaneous injection, 60, 30, or 15 mg/kg body weight for 14–21 days |
Applications | After the treatment, the mice were killed and intestinal tissues were harvested for immunofluorescence staining to determine whether hCFTR protein could be detected. No hCFTR protein was detected in intestinal tissues from untreated mice with hCFTR-specific antiserum. However, strong hCFTR staining was observed at the apical surface of epithelial cells in submucosal glands from mice treated with 60 mg/kg PTC124. Much weaker staining was detected in submucosal glands from mice treated with 30 mg/kg PTC124, whereas no signal could be detected in mice treated with 15 mg/kg PTC124. These results indicate that PTC124 can suppress the G542X mutation and partially restore hCFTR protein expression in Cftr-/- hCFTR-G542X mice. |
Other notes | Please test the solubility of all compounds indoor, and the actual solubility may slightly differ with the theoretical value. This is caused by an experimental system error and it is normal. |
References: [1] Welch E M, Barton E R, Zhuo J, et al. PTC124 targets genetic disorders caused by nonsense mutations. Nature, 2007, 447(7140): 87-91. [2] Du M, Liu X, Welch E M, et al. PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model. Proceedings of the National Academy of Sciences, 2008, 105(6): 2064-2069. | |
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| Cas No. | 775304-57-9 | SDF | Download SDF |
| Synonyms | PTC 124;PTC-124 | ||
| Chemical Name | 3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid | ||
| Canonical SMILES | C1=CC=C(C(=C1)C2=NC(=NO2)C3=CC(=CC=C3)C(=O)O)F | ||
| Formula | C15H9FN2O3 | M.Wt | 284.24 |
| Solubility | ≥10.6mg/mL in DMSO | Storage | Store at -20°C |
| Physical Appearance | A solid | Shipping Condition | Evaluation sample solution : ship with blue ice.All other available size:ship with RT , or blue ice upon request |
| General tips | For obtaining a higher solubility , please warm the tube at 37 ℃ and shake it in the ultrasonic bath for a while.Stock solution can be stored below -20℃ for several months. | ||
PTC124 is a selective inhibitor of nonsense mutations with IC50 value of 0.1μM [1].Nonsense mutation is a point mutation in a sequence of DNA which promotes premature translational termination. Different from missense mutation, nonsense mutation means a single nucleotide is changed and results in the substitution of a different amino acid. It has been reported that some genetic disorders (thalassemia, DMD, CF, Hurler syndrome) are correlated with nonsense mutation [1, 2]. PTC124 is a potent nonsense mutations inhibitor. When tested with human or mdx mice primary muscle cells expressing dystrophin nonsense alleles, 2-8 weeks treatment of PTC124 enhanced the production of dystrophin [1]. In iPSC-derived RPE cells with nonsense mutation c.519C>T (p.R120X), PTC124 treatment restored endogenous, full-length RP2 protein with near 20% [3]. When tested with COS7 cells carrying the nonsense mutation pDsRed-EGFPmtag-Y445X, EGFP transcript level was increased after treated by PTC124 in a dose-dependent manner [2].In mdx mice model, oral administration of PTC124 for 2-8 weeks rescued striated muscle function [1]. In a model of Cftr-/- mice expressing a human CFTR-G542X transgene, s.c. injection or oral administration of PTC124 restored a considerable amount of human (h) CFTR protein and function via suppressing G542X nonsense mutation [4].References: [1]. Welch, E.M., et al., PTC124 targets genetic disorders caused by nonsense mutations. Nature, 2007. 447(7140): p. 87-91.[2]. Shen, Q., P. Guo, and B. Chai, pDsRed-EGFPmtag-, an effective dual fluorescent reporter system for cell-based screens of premature termination codon. Cytotechnology, 2014.[3]. Schwarz, N., et al., Translational read-through of the RP2 Arg120stop mutation in patient iPSC-derived retinal pigment epithelium cells. Hum Mol Genet, 2015. 24(4): p. 972-86.[4]. Du, M., et al., PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model. Proc Natl Acad Sci U S A, 2008. 105(6): p. 2064-9.
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这样的回答可能没分，好和不好都是比较出来的，一群国产的放在一起比总有个性能比较均衡，比较突出性价比好的，
还是没有和说那个厂家的好桂林的呢好像是优利特这个厂家在做，特康的前身呢就是百特，现在的百特呢基本上是在做试剂，这些东西呢。
也搜索的到的，不过答案还是比较接近的，但想知道的是国产那个厂家生产的比较好，而只回答了一半就和介绍了下国产的血球分析仪有那个几个大的厂家，没有和区分那个最好那个第二那个次点，或许一堆鸡蛋里实在挑不出骨头。
具所知呢迈瑞的好点特康实力没有迈瑞好，迈瑞吸引了一批外资在搞研发，桂林的呢好像有个优利特现在出血分析仪，他做的尿分析仪市场的占有还可以，血的反应不是很强烈。
实在是想找个内行的给比较下。
以前用countstar,一块计数板5个孔，后来实验室又换了biorad的TC20，一块板子两个孔，只能计两个数，一盒板子30片500块，价格越来越贵。这样我一个12孔板计个数就要花百来块钱啊。大家都是这么用的？板子是塑料的，有没有什么可以清洗之后重复利用的方法？
