141206-42-0
141206-42-0 structure
- Name: Lucerastat
- Chemical Name: (2R,3S,4R,5S)-1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol
- CAS Number: 141206-42-0
- Molecular Formula: C10H21NO4
- Molecular Weight: 219.27800
- Catalog: Research Areas Metabolic Disease
- Create Date: 2016-03-05 00:47:05
- Modify Date: 2024-01-05 14:35:22
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Lucerastat, the galactose form of Miglustat, is an orally-available inhibitor of glucosylceramide synthase (GCS). Lucerastat has the potential for Fabry disease study[1][2].
| Name | (2R,3S,4R,5S)-1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol |
|---|---|
| Synonyms |
NB-DGJ
UNII-GVS3YDM418 Lucerastat N-Butyl-DGJ N-Bu-DGJ |
| Description | Lucerastat, the galactose form of Miglustat, is an orally-available inhibitor of glucosylceramide synthase (GCS). Lucerastat has the potential for Fabry disease study[1][2]. |
|---|---|
| Related Catalog | |
| 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. |
| 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-/0 and 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. |
| References |
| Molecular Formula | C10H21NO4 |
|---|---|
| Molecular Weight | 219.27800 |
| Exact Mass | 219.14700 |
| PSA | 84.16000 |
| Hazard Codes | Xn |
|---|