1-Deoxygalactonojirimycin (hydrochloride) (Synonyms: DGJ, Migalastat) |
| カタログ番号GC10430 |
1-デオキシガラクトノジリマイシン (塩酸塩) (GR181413A) は、α-ガラクトシダーゼ A (α-Gal A) の強力で競合的な阻害剤であり、IC50 は 0.04 μM for human &8888-Gal A.
Products are for research use only. Not for human use. We do not sell to patients.
Cas No.: 75172-81-5
Sample solution is provided at 25 µL, 10mM.
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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].
References:
[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.
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Animal experiment: |
Mice: Migalastat (GR181413A) hydrochloride is dissolved in drinking water. Age-matched male homozygous TgM, TgM/KO, and nontransgenic (Non-Tg) C57BL/6 mice are supplied drinking water containing DGJ ad libitum. Body weight is monitored weekly during the DGJ treatment period. Average daily DGJ dosage is estimated based on the consumption of drinking water. Mice fed drinking water containing DGJ at 0.05 mM typically received DGJ at approximately 3 mg/kg body weight/day[2]. |
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References: [1]. Asano N, et al. In vitro inhibition and intracellular enhancement of lysosomal alpha-galactosidase A activity in Fabry lymphoblasts by 1-deoxygalactonojirimycin and its derivatives. Eur J Biochem. 2000 Jul;267(13):4179-86. |
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| Cas No. | 75172-81-5 | SDF | |
| 同義語 | DGJ, Migalastat | ||
| Chemical Name | (2R,3S,4R,5S)-2-(hydroxymethyl)-3,4,5-piperidinetriol, monohydrochloride | ||
| Canonical SMILES | O[C@@H]1[C@@H](O)[C@@H](O)[C@@H](CO)NC1.Cl | ||
| Formula | C6H13NO4 • HCl | M.Wt | 199.6 |
| 溶解度 | ≤10mg/ml in DMSO | Storage | Store at -20°C |
| General tips | Please select the appropriate solvent to prepare the stock solution according to the
solubility of the product in different solvents; once the solution is prepared, please store it in
separate packages to avoid product failure caused by repeated freezing and thawing.Storage method
and period of the stock solution: When stored at -80°C, please use it within 6 months; when stored
at -20°C, please use it within 1 month. To increase solubility, heat the tube to 37°C and then oscillate in an ultrasonic bath for some time. |
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| Shipping Condition | Evaluation sample solution: shipped with blue ice. All other sizes available: with RT, or with Blue Ice upon request. | ||
| Prepare stock solution | |||
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1 mg | 5 mg | 10 mg |
| 1 mM | 5.01 mL | 25.0501 mL | 50.1002 mL |
| 5 mM | 1.002 mL | 5.01 mL | 10.02 mL |
| 10 mM | 0.501 mL | 2.505 mL | 5.01 mL |
Step 1: Enter information below (Recommended: An additional animal making an allowance for loss during the experiment)
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μL
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Calculation results:
Working concentration: mg/ml;
Method for preparing DMSO master liquid: mg drug pre-dissolved in μL DMSO ( Master liquid concentration mg/mL, Please contact us first if the concentration exceeds the DMSO solubility of the batch of drug. )
Method for preparing in vivo formulation: Take μL DMSO master liquid, next addμL PEG300, mix and clarify, next addμL Tween 80, mix and clarify, next add μL ddH2O, mix and clarify.
Method for preparing in vivo formulation: Take μL DMSO master liquid, next add μL Corn oil, mix and clarify.
Note: 1. Please make sure the liquid is clear before adding the next solvent.
2. Be sure to add the solvent(s) in order. You must ensure that the solution obtained, in the previous addition, is a clear solution before proceeding to add the next solvent. Physical methods such as vortex, ultrasound or hot water bath can be used to aid dissolving.
3. All of the above co-solvents are available for purchase on the GlpBio website.
Quality Control & SDS
- View current batch:
- Purity: >99.50%
- COA (Certificate Of Analysis)
- SDS (Safety Data Sheet)
- Datasheet
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