5-C-phenethyl-DNJ hydrochloride
5-C-phenethyl-DNJ hydrochloride is an orally active acid α-glucosidase (GAA) activator. 5-C-phenethyl-DNJ hydrochloride can increase GAA activity in various tissues, particularly in the diaphragm. 5-C-phenethyl-DNJ hydrochloride can be used for the research of metabolic disease, such as Pompe disease.
For research use only. We do not sell to patients.
- CAS No.: 2763641-30-9
- Formula: C14H22ClNO4
- Molecular Weight:303.78
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Storage:
Please store the product under the recommended conditions in the Certificate of Analysis.
To place orders, for customer services and technical support, please contact: MedChemExpress USA
Tel: 609-228-6898 E-mail: [email protected] [email protected]
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Biological Activity
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Chemical Information
- Purity & Documentation
- References
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Help & FAQs
Biological Activity
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α‑glucosidase |
5-C-phenethyl-DNJ hydrochloride (Compound 4c) (30 mins) specifically inhibits the activity of recombinant human acid α-glucosidase (rhGAA) with an IC50 of 0.81 μM[1].
5-C-phenethyl-DNJ hydrochloride (100 μM) significantly enhances the stability of rhGAA at neutral pH (7.4), and maintains the structural stability of rhGAA in plasma and non-lysosomal cellular compartments[1].
5-C-phenethyl-DNJ hydrochloride (0.1-100 μM, 30-60 mins) exhibits excellent selectivity for other human glycosidases with IC50 >100 μM for GANAB, GBA1 and GBA2[1].
5-C-phenethyl-DNJ hydrochloride (5-20 μM, 24 h) significantly restores endogenous GAA activity in fibroblasts with Pompe disease carrying different GAA mutations[1].
5-C-phenethyl-DNJ hydrochloride (20 μM, 24 h) enhances the activity and lysosomal delivery of rhGAA in Pompe disease fibroblasts[1].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
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Animal Model:GAA-KO mice models[1]
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Dosage:5 mg/kg, companied with 40 mg/kg rhGAA i.v. at day 3
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Administration:Orally gavage
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Result:Increased the GAA activity by 2 folds in diaphragm (a key tissue for respiratory function).
Increased the GAA activity by 1.5 folds in heart and quadriceps.
Showed no significant difference in liver activity.
Chemical Information
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CAS No. 2763641-30-9
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Molecular Weight 303.78
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Formula C14H22ClNO4
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SMILES
OC[C@@]1([C@H]([C@@H]([C@H](CN1)O)O)O)CCC2=CC=CC=C2.Cl
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Shipping
Room temperature in continental US; may vary elsewhere.
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Storage
Please store the product under the recommended conditions in the Certificate of Analysis.
Purity & Documentation
References
Calculators
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)
5-C-phenethyl-DNJ hydrochloride Related Classifications
HY-178200 Related Classifications
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