| 产品描述： | Migalastat hydrochloride (GR181413A) is a potent and competitive inhibitor of α-galactosidase A (α-Gal A) with an IC50 of 0.04 μM for human α-Gal A. |
| 靶点： |
IC50: 0.04 μM (human α-Gal A); Ki: 0.04 μM (human α-Gal A) |
| 体内研究： |
Fabry disease is an X-linked recessive disorder caused by the deficient activity of α-galactosidase A. Migalastat (oral gavage, 3 mg/kg daily for 4 weeks) increases α-Gal A activity in heart, kidney, spleen, and liver in a dose- and time-dependently in transgenic mice that express human mutant alpha-Gal A (TgM). Migalastat shows the half-life of less than 1 day in all major issues in TgM for 2 weeks pretreatment. Migalastat (oral gavage, 100 mg/kg daily for 28 days) to transgenic mice reduces lyso-Gb3 levels up to 64%, 59%, and 81% in kidney, heart, and skin, respectively |
| 参考文献： |
1. Asano N, et al. In vitro inhibition and intracellular enhancement of lysosomal alpha-galactosidase A activity in Fabry lymphoblasts by 1-deoxygalactonojirimycin and its derivatives. Eur J Biochem. 2000 Jul;267(13):4179-86. 2. Ishii S, et al. Preclinical efficacy and safety of 1-deoxygalactonojirimycin in mice for Fabry disease. J Pharmacol Exp Ther. 2009 Mar;328(3):723-31. 3. Young-Gqamana B, et al. Migalastat HCl reduces globotriaosylsphingosine (lyso-Gb3) in Fabry transgenic mice and in the plasma of Fabry patients. PLoS One. 2013;8(3):e57631. 4. Welford RWD, et al. Glucosylceramide synthase inhibition with lucerastat lowers globotriaosylceramide and lysosome staining in cultured fibroblasts from Fabry patients with different mutation types. Hum Mol Genet. 2018 Oct. 27(19):3392-3403. |
| 溶解性： |
Soluble in H2O |
| 保存条件： |
-80℃（运输条件：冰袋低温运输） |
| 配置溶液浓度参考： |
|
1mg |
5mg |
10mg |
| 1 mM |
5.009 ml |
25.046 ml |
50.093 ml |
| 5 mM |
1.002 ml |
5.009 ml |
10.019 ml |
| 10 mM |
0.501 ml |
2.505 ml |
5.009 ml |
| 50 mM |
0.1 ml |
0.501 ml |
1.002 ml |
|
| 注意： |
部分产品我司仅能提供部分信息，我司不保证所提供信息的权威性，仅供客户参考交流研究之用。 |
上海工商
危险品化学品经营许可证（不带存储）
营业执照(三证合一)
北京