IC50: 40 nM
1-Deoxygalactonojirimycin (hydrochloride) is a competitive inhibitor of α-galactosidase, with the IC50 value of 40 nM . 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 .
 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.
 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.
|Physical Appearance||A crystalline solid|
|Storage||Store at -20°C|
|Formula||C6H13NO4 • HCl|
|Solubility||≤10mg/ml in DMSO|
|Chemical Name||(2R,3S,4R,5S)-2-(hydroxymethyl)-3,4,5-piperidinetriol, monohydrochloride|
|Canonical SMILES||O[[email protected]@H]1[[email protected]@H](O)[[email protected]@H](O)[[email protected]@H](CO)NC1.Cl|
|Shipping Condition||Evaluation sample solution : ship with blue ice.All other available size: ship with RT , or blue ice upon request|
|General tips||For 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.|