N-(n-Butyl)deoxygalactonojirimycin - CAS 141206-42-0
Catalog number:
141206-42-0
Category:
Inhibitor
Not Intended for Therapeutic Use. For research use only.
Molecular Formula:
C10H21NO4
Molecular Weight:
219.28
COA:
Inquire
Targets:
Others
Description:
Lucerastat is a selective α-D-galactosidase inhibitor. It can prevent glucosylceramide biosynthesis both in vitro and in mouse models of lysosome storage disorders through inhibiting the ceramide-specific glucosyltransferase, UDP-glucose ceramide glucosyltransferase (IC50 = 41.4 µM) but it does not affect α-glucosidase I and II or β-glucocerebrosidase (IC50s > 1 mM). In Jul 2016, Actelion completed phase Ib trial in Fabry's disease,but no development was reported for the treatment of Lipid metabolism disorders.
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Purity:
95%
Appearance:
Off-White to Pale Yellow Solid
Synonyms:
(2R,3S,4R,5S)-1-Butyl-2-(hydroxymethyl)-3,4,5-piperidinetriol; NBDGJ;Lucerastat
Solubility:
Methanol, Water
Storage:
-20°C Freezer
MSDS:
Inquire
Application:
lipid storage disorders and Fabry's disease.
Quality Standard:
In-house standard
Shelf Life:
2 month in rt, long time
Quantity:
Milligrams-Grams
Boiling Point:
No Data Available
Melting Point:
123-124°C
InChIKey:
UQRORFVVSGFNRO-XFWSIPNHSA-N
InChI:
1S/C10H21NO4/c1-2-3-4-11-5-8(13)10(15)9(14)7(11)6-12/h7-10,12-15H,2-6H2,1H3/t7-,8+,9+,10-/m1/s1
Canonical SMILES:
OC[C@H]1N(CCCC)C[C@H](O)[C@@H](O)[C@H]1O
Current Developer:
Originator Actelion Pharmaceuticals
1.A bicyclic 1-deoxygalactonojirimycin derivative as a novel pharmacological chaperone for GM1 gangliosidosis.
Takai T1, Higaki K, Aguilar-Moncayo M, Mena-Barragán T, Hirano Y, Yura K, Yu L, Ninomiya H, García-Moreno MI, Sakakibara Y, Ohno K, Nanba E, Ortiz Mellet C, García Fernández JM, Suzuki Y. Mol Ther. 2013 Mar;21(3):526-32. doi: 10.1038/mt.2012.263. Epub 2013 Jan 22.
Lysosomal β-galactosidase (β-Gal) deficiency causes a group of disorders that include neuronopathic GM1 gangliosidosis and non-neuronopathic Morquio B disease. We have previously proposed the use of small molecule ligands of β-Gal as pharmacological chaperones (PCs) for the treatment of GM1 gangliosidosis brain pathology. Although it is still under development, PC therapy has yielded promising preclinical results in several lysosomal diseases. In this study, we evaluated the effect of bicyclic 1-deoxygalactonojirimycin (DGJ) derivative of the sp(2)-iminosugar type, namely 5N,6S-(N'-butyliminomethylidene)-6-thio-1- deoxygalactonojirimycin (6S-NBI-DGJ), as a novel PC for human mutant β-Gal. In vitro, 6S-NBI-DGJ had the ability to inhibit the activity of human β-Gal in a competitive manner and was able to protect this enzyme from heat-induced degradation. Computational analysis supported that the rigid glycone bicyclic core of 6S-NBI-DGJ binds to the active site of the enzyme, with the aglycone N'-butyl substituent, in a precise E-orientation, located at a hydrophobic region nearby.
2.Membrane disruption and cytotoxicity of hydrophobic N-alkylated imino sugars is independent of the inhibition of protein and lipid glycosylation.
Mellor HR1, Platt FM, Dwek RA, Butters TD. Biochem J. 2003 Sep 1;374(Pt 2):307-14.
The N-alkyl moiety of N-alkylated imino sugars is crucial for therapeutic activities of these compounds as inhibitors of glycosphingolipid (GSL) biosynthesis and as antivirals. The improved potency afforded by a long N-alkyl moiety is coincident with increased compound-induced cytotoxicity. Therefore, in the present study, we examined the mechanism of this cytotoxicity in detail. Despite N-butyl-deoxynojirimycin and N-butyl-deoxygalactonojirimycin inhibiting the glycosylation of ceramide to glucosylceramide, ceramide levels did not increase in HL60 cells treated with these compounds. Long-chain N-alkylated imino sugars were toxic to cells at concentrations considerably lower than the critical micellar concentrations for these compounds and consequently did not solubilize radioactively labelled cellular proteins and lipids. However, membrane disruption and cell fragmentation did increase in a concentration- and chain-length-dependent manner.
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CAS 141206-42-0 N-(n-Butyl)deoxygalactonojirimycin

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