Ataluren (PTC124)

目录号:S6003

Ataluren (PTC124) Chemical Structure

Molecular Weight(MW): 284.24

Ataluren (PTC124)选择性诱导核糖体转录通读,但不影响正常的终止密码子,在HEK293细胞中EC50为0.1 μM,可以治疗nonsense mutations (如CFTR无义突变引起的CF)造成的家族遗传性疾病。 Phase 3。

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RMB 799.01 现货
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客户使用Selleck该产品发表文献14篇:

客户使用该产品的8个实验数据:

  • Effect of systemic ataluren treatment on mice with the Pax6Sey+/- phenotype. The black arrowhead indicates the lenticular stalk; the black arrow indicates the cornea; and the asterisk indicates the ciliary margin. WT, Pax6+/+; Mt, Pax6Sey/+; L, lens; r, retina. Original magnification, x5.

    J Clin Invest 2014 124(1), 111-6. Ataluren (PTC124) purchased from Selleck.

    Quantification by qRT-PCR of PCCA (for P1 and P2) and PCCB (P3 and P4) mRNA levels in fibroblast samples untreated or cultured with different concentrations of readthrough drugs. The y-axis represents the results of the relative quantification expressed as percentages. Data represent the mean ?SD of two experiments performed in triplicate. DMSO, dimethyl sulfoxide.

    Hum Mutat 2012 33, 973-80. Ataluren (PTC124) purchased from Selleck.

  • Mesenchymal stromal cells (MSCs) were freshly isolated from bone marrow biopsies and incubated with 2.5-5 μM ataluren for 48, 72, or 96 hours or vehicle alone (DMSO).

    Am J Hematol, 2018, 93(4):527-536. Ataluren (PTC124) purchased from Selleck.

    Tissue-dependent effectiveness of the read-through drug, ataluren (PTC124), in Cln1 R151X mice. Two-month old Cln1 R151X male mice were treated with ataluren (10 mg/kg, i.p.) four times a day for 2 days. Control mice were treated with the vehicle of the drug. Immediately following the last injection on the second day, tissues were collected, and PPT1 enzyme activity was measured in the brain (cerebellum, cortex, striatum/thalamus, brainstem) and peripheral tissues (liver, heart, lung, kidney, skeletal muscle). Ataluren only increased PPT1 activity in the liver and muscle. Columns and bars represent mean ± S.E.M. (eight wild-type and eight Cln1 R151X mice). Statistical significance was determined by unpaired t-test.: *p=0.004 for liver, *p=0.009 for skeletal muscle.

    J Cell Mol Med, 2016, 20(2):381-5 . Ataluren (PTC124) purchased from Selleck.

  • The effect of PTC124, compared with DMSO vehicle, was tested on the subunit c storage in TPP1 patient NPCs. Representative micrograph images are shown of cells immunostained to detect subunit c storage, following 72 h of treatment with 0.5% DMSO or 18 uM PTC124.

    Hum Mol Genet 2014 23(8), 2005-22. Ataluren (PTC124) purchased from Selleck.

    Control and R120X RPE cells were stained for endogenous Gβ1 (green) and the membrane marker WGA (red). Gβ1 plasma membrane localization is disrupted in R120X RPE cells, but restored with treatment of either G418 or PTC124. N = 3 independent experiments with 300 cells per experiment.

    Hum Mol Genet 2014 10.1093/hmg/ddu42. Ataluren (PTC124) purchased from Selleck.

  • Endogenous full-length RP2 cDNA and protein can be restored in R120X fibroblasts by treatment with G418 and PTC124. (A) Treatment of R120X fibroblasts with a single 24 h dose of 500 uM G418 significantly increased RP2 mRNA levels compared with untreated cells. *P ≤ 0.05, values are mean ± 2 SEM, N = 3. (B) Agarose gel showing products of RT-qPCR amplification. GAPDH was used as an amplification control. (C) Treatment of R120X fibroblasts with a single 24 h dose of 750 uM, 500 uM G418 or 10 ug/ml PTC124 significantly increased RP2 protein levels compared with untreated R120X cells. *P ≤ 0.05, values are mean ± 2 SEM, N = 2. (D) Western blot showing increased RP2 protein levels in G418 and PTC124 treated R120X fibroblasts compared with untreated R120X cells.

    Hum Mol Genet 2014 10.1093/hmg/ddu42. Ataluren (PTC124) purchased from Selleck.

    Quantitative RT-PCR analysis of CPT1A in normal and patient fibroblasts treated with various concentration of PTC124 (0, 1, 3 and 5 μM) for 72 hours. The relative contents of mRNA were calculated by using the ΔΔCt method. Expression was normalized to human GAPDH and no significant fold difference was observed in the patient mRNA levels after treatment with PTC124. The normal control cells showed approximately 10-fold higher expression than patient cells. Data are presented as mean; Error bar indicates SEM

    Dr. Lu Tan of Children’s Hospital of Philadelphia. Ataluren (PTC124) purchased from Selleck.

产品安全说明书

CFTR抑制剂选择性比较

生物活性

产品描述 Ataluren (PTC124)选择性诱导核糖体转录通读,但不影响正常的终止密码子,在HEK293细胞中EC50为0.1 μM,可以治疗nonsense mutations (如CFTR无义突变引起的CF)造成的家族遗传性疾病。 Phase 3。
特性 PTC124 具有口服生物有效性,和适当的安全毒性谱。
靶点
CFTR (nonsense mutant) [1]
(HEK293 cells)
体外研究

与 Gentamicin只在较高浓度有活性相比,PTC124是更有效的无义抑制剂,与对照组相比,通读刺激强4-15倍。PTC124(0.01-3 μM)作用于 含LUC-190无义等位基因的HEK293细胞,促进三个无义密码子的通读,这种作用存在剂量依赖性,在UGA通读最高,然后是UAG,再然后是UAA,但是不会抑制多种近端无义密码子。与Gentamicin相似, PTC124在无义密码子后的嘧啶处(尤其是胞嘧啶,C) 活性最高。与稳定细胞系报告实验一致,PTC124 (17 μM) 作用于 Duchenne 肌营养不良症 (DMD) 患者或表达肌萎缩蛋白无义等位基因的mdx小鼠,显著促进肌萎缩蛋白产量。PTC124 选择性促进核糖体提前终止密码子而不是正常终止密码子的通读,即使浓度显著高于达到最大活性所需值。[1]

Cell Data
Cell Lines Assay Type Concentration Incubation Time Formulation Activity Description PMID
BAC_MMA∗EGFP MWDGeY5kfGmxbjDBd5NigQ>? M{DPZ|IxyqEQvF2= NGfyWoc4OsLiaNMg NVLZUXQxTE2VTx?= MoCwdoV{fWy2czDpckBiKHOrZ37p[olk[W62IHnuZ5Jm[XOnIHnuJI1m\GmjbjDw[YFsKG[udX;y[ZNk\W6lZR?= MkPjNlMxPDFzOEm=
Mut−/−MUTStop+/− MnzCSpVv[3Srb36gRZN{[Xl? M{Pwe|IxyqEQvF2= MVi3NuKhcMLi MUnEUXNQ NIjPWmhqdmO{ZXHz[ZMhfGinIHHtc5VvfCCxZtMgUXVVyqCvUl7B MV6yN|A1OTF6OR?=
HEK293T NFexWJJHfW6ldHnvckBCe3OjeR?= MVuxNEDDvWdxbXy= M2D4VmROW09? MVLy[ZN1d3KnczDmeYxtNWynbnf0bEBp[XKvb37pckBiOSBq4pk8PFAhc0SjKTDpckBxNlJ|MWiteJJidnOoZXP0[YQh[2WubIO= MlfQNlMxOjd4NEC=
ML1 MYPGeY5kfGmxbjDBd5NigQ>? M{LrXVMvOy9zMNMg{txO NEezb|c1QCCq Ml3kSG1UVw>? Mm\nbY5kemWjc3XzJGFTW0JiYXP0bZZqfHl? MWWyNlk4OTl3OR?=
ML2 MmmySpVv[3Srb36gRZN{[Xl? M4DKTVMvOy9zMNMg{txO NGi1XmU1QCCq M1m1XWROW09? NHX3R5NqdmO{ZXHz[ZMhSVKVQjDhZ5Rqfmm2eR?= MX:yNlk4OTl3OR?=

... Click to View More Cell Line Experimental Data

体内研究 因为肌萎缩蛋白产量的功能性恢复,PTC124按口服,腹腔注射,或两者联合,处理2-8周,部分缓解 mdx小鼠营养不良肌中的功能性力量不足,结果作用于 趾长伸肌(EDL),对收缩性损伤起到局部保护作用,且显著降低血清肌酸激酶值。[1] PTC124按 ~60 mg/kg剂量皮下或口服处理表达人类CFTR-G542X转基因的Cftr-/-小鼠,抑制G542X 无义突变,这种作用存在剂量依赖性,导致人类 (h)CFTR 蛋白表达和功能的显著恢复,而对无义介导的mRNA 降解 (NMD)或mRNA稳定性的其他方面没有任何影响。PTC124 按 60 mg/kg剂量处理Cftr+/+小鼠,观察到正常肠道跨上皮cAMP刺激的短路电流恢复29%,与Gentamicin相比具有的显著优势。[2]

推荐的实验操作(此推荐来自于公开的文献所以Selleck并不保证其有效性)

动物实验:[2]
+ 展开
  • Animal Models: Cftr-/- hCFTR-G542X 转基因小鼠
  • Formulation: 溶于 DMSO,然后在盐水中稀释
  • Dosages: ~60 mg/kg/day
  • Administration: 皮下注射或口服处理
    (Only for Reference)

溶解度 (25°C)

体外 DMSO 57 mg/mL (200.53 mM)
Water Insoluble
Ethanol Insoluble
体内 从左到右依次将纯溶剂加入产品,现配现用(数据来自Selleck实验检测而非文献):
1% DMSO+30% polyethylene glycol+1% Tween 80
30 mg/mL

* 溶解度检测是由Selleck技术部门检测的,可能会和文献中提供的溶解度有所差异,这是由于生产工艺和批次不同产生的正常现象。请按照顺序依次加入各个纯溶剂。

化学数据

分子量 284.24
化学式

C15H9FN2O3

CAS号 775304-57-9
稳定性 powder
in solvent
别名 N/A

计算器

摩尔浓度计算器

摩尔浓度计算器

本计算器可帮助您计算出特定溶液中溶质的质量、溶液浓度和体积之间的关系,公式为:

质量 (g) = 浓度 (mol/L) x 体积 (L) x 分子量 (g/mol)

摩尔浓度计算公式

  • 质量
    浓度
    体积
    分子量

*在配置溶液时,请务必参考Selleck产品标签上、MSDS / COA(可在Selleck的产品页面获得)批次特异的分子量使用本工具。

稀释计算器

稀释计算器

用本工具协助配置特定浓度的溶液,使用的计算公式为:

开始浓度 x 开始体积 = 最终浓度 x 最终体积

稀释公式

稀释公式一般简略地表示为: C1V1 = C2V2 ( 输入 输出 )

  • C1
    V1
    C2
    V2

在配置溶液时,请务必参考Selleck产品标签上、MSDS / COA(可在Selleck的产品页面获得)批次特异的分子量使用本工具。.

连续稀释计算器方程

  • 连续稀释

  • 计算结果

  • C1=C0/X C1: LOG(C1):
    C2=C1/X C2: LOG(C2):
    C3=C2/X C3: LOG(C3):
    C4=C3/X C4: LOG(C4):
    C5=C4/X C5: LOG(C5):
    C6=C5/X C6: LOG(C6):
    C7=C6/X C7: LOG(C7):
    C8=C7/X C8: LOG(C8):
分子量计算器

分子量计算器

通过输入化合物的化学式来计算其分子量:

总分子量:g/mol

注:化学分子式大小写敏感。C10H16N2O2 c10h16n2o2

摩尔浓度计算器

质量 浓度 体积 分子量
计算

临床试验信息

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT03648827 Recruiting Duchenne Muscular Dystrophy PTC Therapeutics December 21 2018 Phase 2
NCT03648827 Recruiting Duchenne Muscular Dystrophy PTC Therapeutics December 21 2018 Phase 2
NCT03179631 Recruiting Muscular Dystrophy Duchenne|Muscular Dystrophies|Muscular Disorders Atrophic|Muscular Diseases|Musculoskeletal Disease|Neuromuscular Diseases|Nervous System Diseases|Genetic Diseases X-Linked|Genetic Diseases Inborn PTC Therapeutics July 6 2017 Phase 3
NCT03179631 Recruiting Muscular Dystrophy Duchenne|Muscular Dystrophies|Muscular Disorders Atrophic|Muscular Diseases|Musculoskeletal Disease|Neuromuscular Diseases|Nervous System Diseases|Genetic Diseases X-Linked|Genetic Diseases Inborn PTC Therapeutics July 6 2017 Phase 3
NCT03256968 Completed Cystic Fibrosis University of Alabama at Birmingham January 27 2017 Phase 4
NCT03256968 Completed Cystic Fibrosis University of Alabama at Birmingham January 27 2017 Phase 4

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操作手册

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CFTR Signaling Pathway Map

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Cell Lines Assay Type Concentration Incubation Time Formulation Activity Description PMID