N-(n-Butyl)deoxygalactonojirimycin|141206-42-0|(NB-DGJ; N-(n-Butyl)deoxygalactonojirimycin)-陌孚医药/Medlife

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N-(n-Butyl)deoxygalactonojirimycin (Synonyms: NB-DGJ; N-(n-Butyl)deoxygalactonojirimycin)

目录号: PC18440 纯度: ≥98%

CAS No. :141206-42-0

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中文名称	N-(n-Butyl)deoxygalactonojirimycin
中文别名	N -(正丁基)脱氧半乳糖霉素;(2R,3S,4R,5S)-1-丁基-2-(羟基甲基)-3,4,5-哌啶三醇;2-羟基-N,N-二甲基苯甲酰胺
英文名称	N-(n-Butyl)deoxygalactonojirimycin
英文别名	3,4,5-Piperidinetriol,1-butyl-2-(hydroxymethyl)-, (2R,3S,4R,5S)-;N-Butyl-1-deoxygalactonojirimycin;N-(n-Butyl)deoxygalactonojirimycin;Lucerastat;NB-DGJ;N-Bu-DGJ;N-Butyl-DGJ;UNII-GVS3YDM418
Cas No.	141206-42-0
分子式	C₁₀H₂₁NO₄
分子量	219.28
包装储存	Sealed and stored at -20℃, moisture-proof *In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)

生物活性

Lucerastat, the galactose form of Miglustat, is an orally-available inhibitor of glucosylceramide synthase (GCS). Lucerastat has the potential for Fabry disease study.

性状

Solid

体外研究(In Vitro)

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

Medlife has not independently confirmed the accuracy of these methods. They are for reference only.

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.

体内研究(In Vivo)

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

Medlife has not independently confirmed the accuracy of these methods. They are for reference only.

Animal Model:	Fabry mice (Gla 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.

运输条件

Room temperature or refrigerated transportation.

储存方式

Sealed and stored at -20℃, moisture-proof

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

ClinicalTrial

参考文献

[1]. Sanne J van der Veen, et al. Developments in the Treatment of Fabry Disease. J Inherit Metab Dis. 2020 Feb 21. [Information]

[2]. R.W.D. Welford, et al. Lucerastat, an Iminosugar for Substrate Reduction Therapy in Fabry Disease: Preclinical Evidence.

溶解度数据

体外研究:

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

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

配制储备溶液

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

产品不同，其溶解度不同。建议根据产品选择合适的溶剂配制储备溶液；配成溶液后，建议分装保存，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°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)。-80°C 储存时，建议在 6 个月内使用，-20°C 储存时，建议在 1 个月内使用。

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产品使用指南

Data Sheet

1：一般建议：溶解度为Medlife测试数据，可能与文献描述存在差异。这是由于生产工艺和批次不同产生的正常现象。为了使其更好的溶解，请用37℃加热试管并在超声波水浴中震动片刻。不同批次产品溶解度各有差异，仅做参考，具体以实验方案为准。

2：储存条件：粉末-20°C一般情况可以保存3年，溶于溶剂-80°C一般情况可以保存1年。不同产品及不同批次产品可能存在差异，请细致阅读产品信息，并辅助参考相关文献描述。

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