WARNING: This product is for research use only, not for human or veterinary use.
MedKoo CAT#: 555891
Description: ML008, also known as NCGC00092410, is a potent and selective inhibitor of glucocerebrosidase with IC50 of 31 nM.
MedKoo Cat#: 555891
Chemical Formula: C21H27N3O2
Exact Mass: 353.2103
Molecular Weight: 353.466
Elemental Analysis: C, 71.36; H, 7.70; N, 11.89; O, 9.05
Synonym: ML008; ML-008; ML 008; NCGC00092410; NCGC-00092410; NCGC 00092410;
IUPAC/Chemical Name: N-[4-Methyl-2-(4-morpholinyl)-6-quinolinyl]cyclohexanecarboxamide
InChi Key: BXVNPOBNVRBOOV-UHFFFAOYSA-N
InChi Code: InChI=1S/C21H27N3O2/c1-15-13-20(24-9-11-26-12-10-24)23-19-8-7-17(14-18(15)19)22-21(25)16-5-3-2-4-6-16/h7-8,13-14,16H,2-6,9-12H2,1H3,(H,22,25)
SMILES Code: O=C(C1CCCCC1)NC2=CC=C3N=C(N4CCOCC4)C=C(C)C3=C2
Gaucher disease is an autosomal recessive lysosomal storage disorder caused by mutations in the glucocerebrosidase gene. Missense mutations result in reduced enzyme activity that may be due to misfolding, raising the possibility of small-molecule chaperone correction of the defect.
Zheng W, Padia J, Urban DJ, Jadhav A, Goker-Alpan O, Simeonov A, Goldin E, Auld D, LaMarca ME, Inglese J, Austin CP, Sidransky E. Three classes of glucocerebrosidase inhibitors identified by quantitative high-throughput screening are chaperone leads for Gaucher disease. Proc Natl Acad Sci U S A. 2007 Aug 7;104(32):13192-7. doi: 10.1073/pnas.0705637104. Epub 2007 Aug 1. PMID: 17670938; PMCID: PMC1936979.