141206-42-0
中文名稱
N -(正丁基)脫氧半乳糖霉素
英文名稱
N-Butyl-1-deoxygalactonojirimycin
CAS
141206-42-0
分子式
C10H21NO4
分子量
219.28
MOL 文件
141206-42-0.mol
141206-42-0 結(jié)構(gòu)式
基本信息
中文別名
N -(正丁基)脫氧半乳糖霉素 英文別名
OGT-923)ACT-434964
Lucerastat
N-Butyl-1-deoxygalactonojirimycin
Lucerastat (N-Butyldeoxygalactonojirimycin
(2R,3S,4R,5S)-1-Butyl-2-(hydroxyMethyl)-3,4,5-piperidinetriol
3,4,5-Piperidinetriol, 1-butyl-2-(hydroxymethyl)-, (2R,3S,4R,5S)-
(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
物理化學(xué)性質(zhì)
熔點(diǎn)123-124°C
沸點(diǎn)394.7±42.0 °C(Predicted)
密度1.234±0.06 g/cm3(Predicted)
儲(chǔ)存條件Hygroscopic, -20°C Freezer, Under Inert Atmosphere
溶解度DMF: 20 mg/mL; DMSO: 30 mg/mL; Ethanol: 5 mg/mL; PBS (pH 7.2): 10 mg/mL
酸度系數(shù)(pKa)13.72±0.70(Predicted)
形態(tài)結(jié)晶固體
顏色White to yellow
穩(wěn)定性吸濕性
常見問題列表
生物活性
Lucerasta 是Miglustat 的半乳糖形式，是具有口服活性的葡萄糖酰神經(jīng)酰胺合成酶 (GCS) 的抑制劑。Lucerastat 有用于Fabry 疾病研究的潛能。體外研究
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. |
體內(nèi)研究
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. |