Lucerastat

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Lucerastat

本产品不向个人销售，仅用作科学研究，不用于任何人体实验及非科研性质的动物实验。

CAS NO:

141206-42-0

NB-DGJ
N-(n-Butyl)deoxygalactonojirimycin

Lucerasta 是Miglustat 的半乳糖形式，是具有口服活性的葡萄糖酰神经酰胺合成酶(GCS)的抑制剂。Lucerastat 有用于Fabry 疾病研究的潜能。

生物活性

Lucerastat, the galactose form of Miglustat, is an orally-available inhibitor ofglucosylceramide synthase (GCS). Lucerastat has the potential for Fabry disease study^[1]^[2].

体外研究
(In Vitro)

Fabry patient-derived fibroblasts with the genotypes R301G (residual -GalA activity; 20%) R220X (<3%) and W162X (<1%).

Cell Viability Assay^[2].

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.

体内研究
(In Vivo)

Lucerastat (1200 mg/kg/day food admix), a GCS inhibitor, reduces Gb3 in the absence of residual ?-GalA activity^[2].

Animal Model:	Fabry mice (Gla^-/0and Gla^-/-, n = 5 or 6 for each gender)^[2].
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.

Clinical Trial

分子量

219.28

性状

Solid

Formula

C₁₀H₂₁NO₄

CAS 号

141206-42-0

运输条件

Room temperature in continental US; may vary elsewhere.

储存方式

-20°C, sealed storage, away from moisture

*In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)

溶解性数据

In Vitro:

H₂O : 24 mg/mL(109.45 mM;ultrasonic and warming and heat to 80℃)

DMSO : 22 mg/mL(100.33 mM;ultrasonic and warming and heat to 80℃)

配制储备液

浓度溶剂体积质量

1 mg

5 mg

10 mg

1 mM	4.5604 mL	22.8019 mL	45.6038 mL
5 mM	0.9121 mL	4.5604 mL	9.1208 mL
10 mM	0.4560 mL	2.2802 mL	4.5604 mL

请根据产品在不同溶剂中的溶解度选择合适的溶剂配制储备液；一旦配成溶液，请分装保存，避免反复冻融造成的产品失效。
储备液的保存方式和期限：-80℃, 6 months; -20℃, 1 month (sealed storage, away from moisture)。-80℃ 储存时，请在 6 个月内使用，-20℃ 储存时，请在 1 个月内使用。