Target Validation Information
TTD ID	T84173
Target Name	Glucosylceramidase (GBA)
Type of Target	Successful
Drug Potency against Target	(2R,3S,4S,5R)-2-hexylpiperidine-3,4,5-triol	Drug Info	IC50 = 19 nM	[3]
	(2R,3S,4S,5R)-2-nonylpiperidine-3,4,5-triol	Drug Info	Ki = 2.2 nM	[3]
	(2R,3S,4S,5R)-2-propylpiperidine-3,4,5-triol	Drug Info	IC50 = 560 nM	[3]
	1,5-Dideoxy-1,5-imino-D-xylitol	Drug Info	IC50 = 2300 nM	[1]
	L-Isofagomine	Drug Info	IC50 = 9000 nM	[1]
	N-(Propylamide-acetophenone)-1-deoxynojirimycin	Drug Info	IC50 = 1650 nM	[2]
	N-(Propylamide-benzophenone)-1-deoxynojirimycin	Drug Info	IC50 = 350 nM	[2]
Action against Disease Model	Isofagomine tartrate	Drug Info	Isofagomine tartrate increases L444P GCase activity in Gaucher patient-derived cells	[4]
References
REF 1	In vitro inhibition of glycogen-degrading enzymes and glycosidases by six-membered sugar mimics and their evaluation in cell cultures. Bioorg Med Chem. 2008 Aug 1;16(15):7330-6.
REF 2	Nanomolar affinity, iminosugar-based chemical probes for specific labeling of lysosomal glucocerebrosidase. Bioorg Med Chem. 2010 Jan 1;18(1):267-73.
REF 3	Synthesis of new beta-1-C-alkylated imino-L-iditols: A comparative study of their activity as beta-glucocerebrosidase inhibitors. Bioorg Med Chem. 2010 Apr 1;18(7):2645-50.
REF 4	The pharmacological chaperone isofagomine increases the activity of the Gaucher disease L444P mutant form of beta-glucosidase. FEBS J. 2010 Apr;277(7):1618-38.

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