Exaluren disulfate
Based on 2 publication(s) in Google Scholar
Exaluren (ELX-02; NB-124) disulfate is an synthetic eukaryotic ribosome-selective glycoside that induces read-through of nonsense mutations, resulting in normally localized full-length functional proteins. Exaluren disulfate is used for the research of cystic fibrosis caused by nonsense mutations.
For research use only. We do not sell to patients.
- Purity: ≥98.0%
- CAS No.: 2244622-33-9
- Formula: C19H42N4O18S2
- Molecular Weight:678.68
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Storage:
-20°C, stored under nitrogen
* In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen)
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
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Solvent & Solubility
- Purity & Documentation
- References
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Help & FAQs
Publications Citing Use of MedChemExpress (MCE) Exaluren disulfate
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Customer Validation & Images
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Bio/Physico-chemical Assay
All DNA/RNA Synthesis Isoforms
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Biological Activity
Exaluren disulfate induces significant premature stop codon read-through with maintained native stop codon fidelity in DMS-114 cells, increasing p53 protein expression and reducing nonsense-mediated mRNA decay of TP53 mRNA[1].
Exaluren disulfate exhibits consistent nonsense mutation read-through activity and efficacy in human bronchial epithelial cells, Fischer rat thyroid cells, and a dual luciferase cystic fibrosis model[1].
Exaluren disulfate induces read-through of the CTNS W138X nonsense allele in human cystinosis fibroblasts, increasing cystinosin protein and CTNS mRNA levels while reducing half-cystine levels[1].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
Exaluren (10 mg/kg; s.c.; 7 injections over a 3-week period) disulfate reduces kidney cell cystine levels by 30% in CTNSY226X nonsense mutant mice without overt renal toxicity[1].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
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Animal Model:CF mouse model expressing a human CFTRG542X transgene[1]
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Dosage:30, 60 mg/kg
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Administration:s.c.; daily; 14 days
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Result:Achieved CFTR functional rescue superior to gentamicin and exhibited favorable pharmacodynamic properties.
Chemical Information
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CAS No. 2244622-33-9
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Appearance Solid
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Molecular Weight 678.68
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Formula C19H42N4O18S2
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Color Yellow to brown
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SMILES
OS(=O)(O)=O.C[C@@H](O)[C@@]([C@@H](O)[C@H](O)[C@H]1N)([H])O[C@]1([H])O[C@H]([C@H](C[C@@H](N)[C@@H]2O)N)[C@]2([H])O[C@@H]3O[C@@]([C@@H](N)C)([H])[C@@H](O)[C@H]3O.OS(=O)(O)=O
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Synonyms
ELX-02 disulfate; NB-124 disulfate
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Shipping
Room temperature in continental US; may vary elsewhere.
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Storage
-20°C, stored under nitrogen
* In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen)
Publications (2)
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Journal Impact Factor
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Most Recent
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Mol Ther
Use of 2,6-diaminopurine as a potent suppressor of UGA premature stop codons in cystic fibrosis. [Abstract]2023 Apr 5;31(4):970-985. PMID: 36641622
Exaluren disulfate purchased from MedChemExpress. Usage Cited in: Mol Ther. 2023 Apr 5;31(4):970-985. [Abstract]
Exaluren disulfate (ELX-02; 20, 40, 80, 160 μM; 24, 48 h) caused slight organoid swelling.
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Genet Med Open
CFTR rescue in W1282X cystic fibrosis patient-derived intestinal organoids (PDIOs) mediated by translational readthrough-inducing drugs (TRIDs). [Abstract]2025 Nov 17:4:103472. PMID: 41492359
Solvent & Solubility
H2O : 100 mg/mL (147.34 mM; Need ultrasonic)
DMSO : < 1 mg/mL (insoluble or slightly soluble)
Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month (stored under nitrogen). When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.
* Note: If you choose water as the stock solution, please dilute it to the working solution, then filter and sterilize it with a 0.22 μm filter before use.
Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month (stored under nitrogen). When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.
* Note: If you choose water as the stock solution, please dilute it to the working solution, then filter and sterilize it with a 0.22 μm filter before use.
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)
For the following dissolution methods, please prepare the working solution directly:
It is recommended to prepare fresh solutions and use them promptly within a short period of time.
The percentages shown for the solvents indicate their volumetric ratio in the final prepared solution. If precipitation or phase separation occurs during preparation, heat and/or sonication can be used to aid dissolution.
Add each solvent one by one: PBS
Solubility: 100 mg/mL (147.34 mM); Clear solution; Need ultrasonic
Purity & Documentation
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Data Sheet (283 KB)
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SDS (252 KB)
- English - EN (252 KB)
- Français - FR (252 KB)
- Deutsch - DE (252 KB)
- Norwegian - NO (252 KB)
- Español - ES (252 KB)
- Swedish - SV (252 KB)
- Italian - IT (252 KB)
- Korean - KR (252 KB)
- Portuguese - PT (252 KB)
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Handling Instructions (2659 KB)
References
[1]. Kerem E, et al. ELX-02: an investigational read-through agent for the treatment of nonsense mutation-related genetic disease. Expert Opin Investig Drugs. 2020;29(12):1347-1354. [Content Brief]
[2]. Leubitz A, et al. Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses of ELX-02, a Potential Treatment for Genetic Disorders Caused by Nonsense Mutations, in Healthy Volunteers. Clin Pharmacol Drug Dev. 2019 Jan 16. [Content Brief]
Complete Stock Solution Preparation Table
Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month (stored under nitrogen). When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.
| Optional Solvent | Concentration Solvent Mass | 1 mg | 5 mg | 10 mg | 25 mg |
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| H2O | 1 mM | 1.4734 mL | 7.3672 mL | 14.7345 mL | 36.8362 mL |
| 5 mM | 0.2947 mL | 1.4734 mL | 2.9469 mL | 7.3672 mL | |
| 10 mM | 0.1473 mL | 0.7367 mL | 1.4734 mL | 3.6836 mL | |
| 15 mM | 0.0982 mL | 0.4911 mL | 0.9823 mL | 2.4557 mL | |
| 20 mM | 0.0737 mL | 0.3684 mL | 0.7367 mL | 1.8418 mL | |
| 25 mM | 0.0589 mL | 0.2947 mL | 0.5894 mL | 1.4734 mL | |
| 30 mM | 0.0491 mL | 0.2456 mL | 0.4911 mL | 1.2279 mL | |
| 40 mM | 0.0368 mL | 0.1842 mL | 0.3684 mL | 0.9209 mL | |
| 50 mM | 0.0295 mL | 0.1473 mL | 0.2947 mL | 0.7367 mL | |
| 60 mM | 0.0246 mL | 0.1228 mL | 0.2456 mL | 0.6139 mL | |
| 80 mM | 0.0184 mL | 0.0921 mL | 0.1842 mL | 0.4605 mL | |
| 100 mM | 0.0147 mL | 0.0737 mL | 0.1473 mL | 0.3684 mL |
* Note: If you choose water as the stock solution, please dilute it to the working solution, then filter and sterilize it with a 0.22 μm filter before use.
Exaluren disulfate Related Classifications
HY-114231B Related Classifications
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