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Lucerastat

CAS No. 141206-42-0

Lucerastat ( N-Butyldeoxygalactonojirimycin | ACT-434964 | OGT-923 )

产品货号. M11715 CAS No. 141206-42-0

一种半乳糖类似物,是一种 UDP-葡萄糖神经酰胺葡萄糖基转移酶 (UGCG/GCS) 抑制剂 (IC50=41.4 uM)。

纯度: >98% (HPLC)

COA Datasheet HNMR HPLC MSDS Handing Instructions
规格 价格/人民币 库存 数量
5MG ¥4179 有现货
500MG 获取报价 有现货
1G 获取报价 有现货

生物学信息

  • 产品名称
    Lucerastat
  • 注意事项
    本公司产品仅用于科研实验,不得用于人体或动物的临床与诊断
  • 产品简述
    一种半乳糖类似物,是一种 UDP-葡萄糖神经酰胺葡萄糖基转移酶 (UGCG/GCS) 抑制剂 (IC50=41.4 uM)。
  • 产品描述
    A galactose analogue that is an UDP-glucose ceramide glucosyltransferase (UGCG/GCS) inhibitor (IC50=41.4 uM); prevents glucosylceramide biosynthesis both in vitro and in mouse models of lysosome storage disorders, without affecting α-glucosidase I and II or β-glucocerebrosidase (IC50>1 mM); shows potential as substrate reduction therapy for glycolipid storage disorders such as Gaucher disease and Fabry disease.Other Indication Phase 1 Clinical.
  • 体外实验
    Fabry patient-derived fibroblasts with the genotypes R301G (residual -GalA activity; 20%) R220X (<3%) and W162X (<1%). Cell Viability Assay.Cell Line:Fabry patient-derived fibroblasts with the genotypes R301G (residual-GalA activity; 20%) R220X (<3%) and W162X (<1%). Concentration:Incubation Time:9 days.Result:Dose-dependently inhibited GCS, reducing glucosylceramide and increasing sphingomyelin.
  • 体内实验
    Lucerastat (1200 mg/kg/day food admix), a GCS inhibitor, reduces Gb3 in the absence of residual-GalA activity. Animal Model:Fabry mice (Gla-/0 and Gla-/-, n = 5 or 6 for each gender).Dosage:1200 mg/kg/day food admix.Administration:Food admix for 20 weeks.Result:Reduced lipid storage in two major organs affected by FD: mean Gb3 in the kidneys (-33%, p<0.01). and α-galactose- terminated glycosphingolipids in the dorsal root ganglia (-48%, p<0.05). In the liver of the Fabry mice, mean glucosylceramide (GlcCer (24:0)) was reduced (-59%, p<0.001) in addition to Gb3 (24:1) (-37%, p<0.05) demonstrating substrate reduction through GCS inhibition.
  • 同义词
    N-Butyldeoxygalactonojirimycin | ACT-434964 | OGT-923
  • 通路
    Others
  • 靶点
    Other Targets
  • 受体
    Other Targets
  • 研究领域
    Other Indications
  • 适应症
    Other Disease

化学信息

  • CAS Number
    141206-42-0
  • 分子量
    219.281
  • 分子式
    C10H21NO4
  • 纯度
    >98% (HPLC)
  • 溶解度
    In Vitro:?H2O : 24 mg/mL (109.45 mM)
  • SMILES
    O[C@H]1[C@@H](CO)N(CCCC)C[C@H](O)[C@H]1O
  • 化学全称
    (2R,3S,4R,5S)-1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol

运输与储存

  • 储存条件
    (-20℃)
  • 运输条件
    With Ice Pack
  • 稳定性
    ≥ 2 years

参考文献

1. Platt FM, et al. J Biol Chem. 1994 Oct 28;269(43):27108-14. 2. Andersson U, et al. Biochem Pharmacol. 2000 Apr 1;59(7):821-9. 3. Kasperzyk JL, et al. J Neurochem. 2004 May;89(3):645-53.
产品手册
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