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1-Deoxygalactonojirimycin (hydrochloride)

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Catalog No.
α-galactosidase inhibitor
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Target: α-galactosidase

IC50: 40 nM

1-Deoxygalactonojirimycin (hydrochloride) is a competitive inhibitor of α-galactosidase, with the IC50 value of 40 nM [1]. 1-Deoxygalactonojirimycin (hydrochloride) is used to promote lysosomal delivery of unstable proteins in lysosomal storage disorders, like Fabry disease. Fabry disease is a rare genetic lysosomal storage disease, inherited in an X-linked manner which is caused by mutations in the α-galactosidase gene that commonly lead to enzyme instability, misfolding, and degradation.

In Vivo: In transgenic mice expressed human mutant α-galactosidase A, treatment with 1-Deoxygalactonojirimycin at a dosage of approximately 3 mg/kg body weight/day could significantly reduce globotriaosylceramide storage in the kidney. Besides, no abnormality of blood chemistry and pathological tissue damage was found in mice treated with 1-Deoxygalactonojirimycin at about 30 mg/kg body weight/day for 9 weeks [2].

[1] Asano N, Ishii S, Kizu H, et al.  In vitro inhibition and intracellular enhancement of lysosomal α‐galactosidase A activity in Fabry lymphoblasts by 1‐deoxygalactonojirimycin and its derivatives[J]. FEBS Journal, 2000, 267(13): 4179-4186.
[2] Ishii S, Chang H, Yoshioka H, et al.  Preclinical Efficacy and Safety of 1-Deoxygalactonojirimycin in Mice for Fabry Disease[J]. Journal of Pharmacology and Experimental Therapeutics, 2009, 328(3): 723-731.

Chemical Properties

Physical AppearanceA crystalline solid
StorageStore at -20°C
Cas No.75172-81-5
FormulaC6H13NO4 • HCl
Solubility≤10mg/ml in DMSO
Chemical Name(2R,3S,4R,5S)-2-(hydroxymethyl)-3,4,5-piperidinetriol, monohydrochloride
SDFDownload SDF
Canonical SMILESO[[email protected]@H]1[[email protected]@H](O)[[email protected]@H](O)[[email protected]@H](CO)NC1.Cl
Shipping ConditionEvaluation sample solution : ship with blue ice.All other available size: ship with RT , or blue ice upon request
General tipsFor obtaining a higher solubility , please warm the tube at 37 ℃ and shake it in the ultrasonic bath for a while.Stock solution can be stored below -20℃ for several months.

Quality Control

Quality Control & MSDS

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Chemical structure

1-Deoxygalactonojirimycin (hydrochloride)