N -(正丁基)脱氧半乳糖霉素
| 中文名称 | N -(正丁基)脱氧半乳糖霉素 |
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| 中文同义词 | N -(正丁基)脱氧半乳糖霉素;化合物 T32923;米加司他杂质1;化合物 LUCERASTAT;芦舍司他;化合物:N-(n-Butyl)deoxygalactonojirimycin;化合物：N-(n-Butyl)deoxygalactonojirimycin |
| 英文名称 | N-Butyl-1-deoxygalactonojirimycin |
| 英文同义词 | N-Butyl-1-deoxygalactonojirimycin;(2R,3S,4R,5S)-1-(But-1-yl)-2-(hydroxymethyl)piperidine-3,4,5-triol;N-(But-1-yl)deoxygalactonojirimycin, (2R,3S,4R,5S)-1-(But-1-yl)-2-(hydroxymethyl)-3,4,5-trihydroxypiperidine;(2R,3S,4R,5S)-1-Butyl-2-(hydroxyMethyl)-3,4,5-piperidinetriol;Lucerastat;ACT-434964;Lucerastat (N-Butyldeoxygalactonojirimycin;OGT-923) |
| CAS号 | 141206-42-0 |
| 分子式 | C10H21NO4 |
| 分子量 | 219.28 |
| EINECS号 | |
| 相关类别 | Glycosidase Inhibitors;Inhibitors |
| Mol文件 | 141206-42-0.mol |
| 结构式 |  |
N -(正丁基)脱氧半乳糖霉素 性质
| 熔点 | 123-124°C |
|---|---|
| 沸点 | 394.7±42.0 °C(Predicted) |
| 密度 | 1.234±0.06 g/cm3(Predicted) |
| 储存条件 | Hygroscopic, -20°C Freezer, Under Inert Atmosphere |
| 溶解度 | DMF：20mg/mL；二甲基亚砜：30mg/mL；乙醇：5mg/mL； PBS（pH 7.2）：10 mg/mL |
| 酸度系数(pKa) | 13.72±0.70(Predicted) |
| 形态 | 结晶固体 |
| 颜色 | 白色至黄色 |
| 稳定性 | 吸湿性 |
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。
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. |
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. |
安全信息
| 海关编码 | 2933399990 |
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