2. Metabolic Enzyme/Protease
  3. Glycosidase
  4. 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.

5-C-phenethyl-DNJ hydrochloride

5-C-phenethyl-DNJ hydrochloride Chemical Structure

CAS No. : 2763641-30-9

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5-C-phenethyl-DNJ hydrochloride Related Antibodies

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Description

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[1].

IC50 & Target[1]

α‑glucosidase

 

In Vitro

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.

5-C-phenethyl-DNJ hydrochloride Related Antibodies
In Vivo

5-C-phenethyl-DNJ (hydrochloride) (Compound 4c) (5 mg/kg, p.o., daily for 4 days) significantly enhances the activity of rhGAA in tissues of GAA-KO mice[1].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

Animal Model: GAA-KO mice models[1]
Dosage: 5 mg/kg, companied with 40 mg/kg rhGAA i.v. at day 3
Administration: Orally gavage
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.
Molecular Weight

303.78

Formula

C14H22ClNO4
CAS No.
SMILES

OC[C@@]1([C@H]([C@@H]([C@H](CN1)O)O)O)CCC2=CC=CC=C2.Cl
Shipping

Room temperature in continental US; may vary elsewhere.
Storage

Please store the product under the recommended conditions in the Certificate of Analysis.
Purity & Documentation
References
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5-C-phenethyl-DNJ hydrochloride Related Classifications

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Help & FAQs
  • Do most proteins show cross-species activity?

    Species cross-reactivity must be investigated individually for each product. Many human cytokines will produce a nice response in mouse cell lines, and many mouse proteins will show activity on human cells. Other proteins may have a lower specific activity when used in the opposite species.

Keywords:

5-C-phenethyl-DNJ2763641-30-9Glycosidaseacid α-glucosidaseGAAPompe diseaseFibroblastsInhibitorinhibitorinhibit

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