| Size | Price | Stock | Qty |
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| 5mg |
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| 10mg |
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| 50mg |
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| Targets |
NN-DNJ is a potent inhibitor of acid α-glucosidase and α-1,6-glucosidase. Its primary targets are these key enzymes involved in glycogen degradation. By inhibiting acid α-glucosidase, NN-DNJ can prevent the breakdown of glycogen in the lysosomes. It also acts as a chemical chaperone, a property that is being explored for its potential to correct misfolding of proteins, including mutant forms of acid α-glucosidase that cause Pompe disease. Its mechanism involves binding to the active site of the target glucosidases, thereby blocking their catalytic activity.
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| ln Vitro |
In vitro, NN-DNJ is a potent inhibitor of acid α-glucosidase and α-1,6-glucosidase, with IC50 values of 0.42 μM and 8.4 μM, respectively. These values demonstrate its high potency, particularly against acid α-glucosidase. This inhibitory activity prevents the breakdown of glycogen in cellular models. Additionally, its function as a chemical chaperone has been demonstrated in vitro, where it can bind to and stabilize the conformation of mutant enzymes, facilitating their proper trafficking to the lysosome.
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| ln Vivo |
In vivo, NN-DNJ (250 mg/kg per day) increases hepatic glycogen levels in fasted mice and gastrocnemius muscle glycogen levels in both fasted and fed mice. This demonstrates its ability to inhibit glycogen breakdown systemically. By inhibiting liver glycogen breakdown, it increases hepatic glycogen stores. These in vivo effects are consistent with its mechanism as a glucosidase inhibitor. Its potential as a chemical chaperone in vivo is also being investigated for the treatment of lysosomal storage disorders like Pompe disease.
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| Enzyme Assay |
In cell-free enzyme assays, the inhibitory activity of NN-DNJ against its targets can be measured using standard glucosidase assays. For acid α-glucosidase, the assay involves incubating varying concentrations of NN-DNJ with a fixed concentration of the purified enzyme and a fluorogenic substrate, such as 4-methylumbelliferyl-α-D-glucopyranoside. The rate of substrate hydrolysis, which is proportional to enzyme activity, is monitored by fluorescence. The IC50 value is calculated from a dose-response curve. Similarly, the inhibition of α-1,6-glucosidase can be measured using a specific substrate and protocol.
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| Cell Assay |
For in vitro cellular assays, the effect of NN-DNJ on glycogen breakdown can be studied in cultured cells, such as hepatocytes or muscle cells. Cells are treated with NN-DNJ for a defined period, and the glycogen content is measured using a colorimetric or enzymatic assay. The reduction in glycogen degradation in the presence of the inhibitor is a measure of its activity. Its function as a chemical chaperone can be assessed in cells expressing a mutant form of acid α-glucosidase, where its ability to increase the enzyme's activity and proper lysosomal localization is measured.
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| Animal Protocol |
For in vivo studies, NN-DNJ is typically administered orally or intraperitoneally to animal models. In mice, its effect on glycogen levels is assessed by treating animals with the compound and then measuring glycogen content in the liver and muscle tissues using biochemical assays. For studying its potential in Pompe disease, it can be administered to a mouse model of the disease, and endpoints include glycogen accumulation in tissues, enzyme activity, and histological analysis of muscle pathology.
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| ADME/Pharmacokinetics |
NN-DNJ (C15H31NO4) has a molecular weight of 289.41 g/mol. Its CAS number is 81117-35-3. It is typically supplied as a powder. It is soluble in DMSO and water. For in vitro studies, stock solutions are prepared in DMSO or water. For in vivo administration, it can be formulated in saline or other suitable buffers. Storage is recommended at -20°C, protected from light. Its purity is typically >98% for research use.
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| Toxicity/Toxicokinetics |
No detailed toxicity data is publicly available. As a glucosidase inhibitor, its safety profile would need to be established through standard toxicological studies. However, as a derivative of 1-deoxynojirimycin, a class of compounds that has been studied for various therapeutic applications, it is likely to have a manageable toxicity profile at therapeutic doses.
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| References | |
| Additional Infomation |
N-Nonyldeoxynojirimycin is a hydroxypiperidine compound, derived from deoxynojirimycin (dovoglustat) where the amino hydrogen is replaced by a nonyl group. It possesses multiple functions, including as an EC 3.2.1.45 (glucosylceramidinase) inhibitor, an EC 3.2.1.20 (α-glucosidase) inhibitor, and an antiviral drug. It is a hydroxypiperidine and tertiary amine compound, functionally related to dovoglustat.
NN-DNJ is a research-grade compound and is not approved for any therapeutic use. It serves primarily as a valuable pharmacological tool for studying glycogen metabolism, glucosidase function, and as a chemical chaperone for lysosomal storage disorders. Its potent inhibitory activity against acid α-glucosidase makes it a key compound in Pompe disease research. Its mechanism of action involves the inhibition of glycogen-degrading enzymes, leading to increased glycogen accumulation. No clinical trials have been reported. |
| Molecular Formula |
C15H31NO4
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|---|---|
| Molecular Weight |
289.41094
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| Exact Mass |
289.225
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| CAS # |
81117-35-3
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| PubChem CID |
501640
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| Appearance |
White to off-white solid powder
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| Density |
1.112g/cm3
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| Boiling Point |
451.7ºC at 760 mmHg
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| Melting Point |
101-102ºC
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| Flash Point |
224.5ºC
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| Index of Refraction |
1.522
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| LogP |
0.434
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| Hydrogen Bond Donor Count |
4
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| Hydrogen Bond Acceptor Count |
5
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| Rotatable Bond Count |
9
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| Heavy Atom Count |
20
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| Complexity |
252
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| Defined Atom Stereocenter Count |
4
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| SMILES |
CCCCCCCCCN1C[C@@H]([C@H]([C@@H]([C@H]1CO)O)O)O
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| InChi Key |
FTSCEGKYKXESFF-LXTVHRRPSA-N
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| InChi Code |
InChI=1S/C15H31NO4/c1-2-3-4-5-6-7-8-9-16-10-13(18)15(20)14(19)12(16)11-17/h12-15,17-20H,2-11H2,1H3/t12-,13+,14-,15-/m1/s1
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| Chemical Name |
(2R,3R,4R,5S)-2-(hydroxymethyl)-1-nonylpiperidine-3,4,5-triol
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| HS Tariff Code |
2934.99.9001
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| Storage |
Powder -20°C 3 years 4°C 2 years In solvent -80°C 6 months -20°C 1 month Note: Please store this product in a sealed and protected environment, avoid exposure to moisture. |
| Shipping Condition |
Room temperature (This product is stable at ambient temperature for a few days during ordinary shipping and time spent in Customs)
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| Solubility (In Vitro) |
DMSO : ~50 mg/mL (~172.77 mM)
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| Solubility (In Vivo) |
Note: Listed below are some common formulations that may be used to formulate products with low water solubility (e.g. < 1 mg/mL), you may test these formulations using a minute amount of products to avoid loss of samples.
Injection Formulations
Injection Formulation 1: DMSO : Tween 80： Saline = 10 : 5 : 85 (i.e. 100 μL DMSO stock solution → 50 μL Tween 80 → 850 μL Saline)(e.g. IP/IV/IM/SC) *Preparation of saline: Dissolve 0.9 g of sodium chloride in 100 mL ddH ₂ O to obtain a clear solution. Injection Formulation 2: DMSO : PEG300 ：Tween 80 : Saline = 10 : 40 : 5 : 45 (i.e. 100 μL DMSO → 400 μLPEG300 → 50 μL Tween 80 → 450 μL Saline) Injection Formulation 3: DMSO : Corn oil = 10 : 90 (i.e. 100 μL DMSO → 900 μL Corn oil) Example: Take the Injection Formulation 3 (DMSO : Corn oil = 10 : 90) as an example, if 1 mL of 2.5 mg/mL working solution is to be prepared, you can take 100 μL 25 mg/mL DMSO stock solution and add to 900 μL corn oil, mix well to obtain a clear or suspension solution (2.5 mg/mL, ready for use in animals). View More
Injection Formulation 4: DMSO : 20% SBE-β-CD in saline = 10 : 90 [i.e. 100 μL DMSO → 900 μL (20% SBE-β-CD in saline)] Oral Formulations
Oral Formulation 1: Suspend in 0.5% CMC Na (carboxymethylcellulose sodium) Oral Formulation 2: Suspend in 0.5% Carboxymethyl cellulose Example: Take the Oral Formulation 1 (Suspend in 0.5% CMC Na) as an example, if 100 mL of 2.5 mg/mL working solution is to be prepared, you can first prepare 0.5% CMC Na solution by measuring 0.5 g CMC Na and dissolve it in 100 mL ddH2O to obtain a clear solution; then add 250 mg of the product to 100 mL 0.5% CMC Na solution, to make the suspension solution (2.5 mg/mL, ready for use in animals). View More
Oral Formulation 3: Dissolved in PEG400 (Please use freshly prepared in vivo formulations for optimal results.) |
| Preparing Stock Solutions | 1 mg | 5 mg | 10 mg | |
| 1 mM | 3.4553 mL | 17.2765 mL | 34.5531 mL | |
| 5 mM | 0.6911 mL | 3.4553 mL | 6.9106 mL | |
| 10 mM | 0.3455 mL | 1.7277 mL | 3.4553 mL |
*Note: Please select an appropriate solvent for the preparation of stock solution based on your experiment needs. For most products, DMSO can be used for preparing stock solutions (e.g. 5 mM, 10 mM, or 20 mM concentration); some products with high aqueous solubility may be dissolved in water directly. Solubility information is available at the above Solubility Data section. Once the stock solution is prepared, aliquot it to routine usage volumes and store at -20°C or -80°C. Avoid repeated freeze and thaw cycles.
Calculation results
Working concentration： mg/mL；
Method for preparing DMSO stock solution： mg drug pre-dissolved in μL DMSO (stock solution 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 stock solution, next add μL PEG300, mix and clarify, next addμL Tween 80, mix and clarify, next add μL ddH2O，mix and clarify.
(1) Please be sure that the solution is clear before the addition of next solvent. Dissolution methods like vortex, ultrasound or warming and heat may be used to aid dissolving.
(2) Be sure to add the solvent(s) in order.
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