Exaluren sulfate
Based on 2 publication(s) in Google Scholar
Exaluren (ELX-02; NB-124) sulfate is an synthetic eukaryotic ribosome-selective glycoside that induces read-through of nonsense mutations, resulting in normally localized full-length functional proteins. Exaluren sulfate is used for the research of cystic fibrosis caused by nonsense mutations.
For research use only. We do not sell to patients.
- CAS No.: 1375073-94-1
- Formula: C19H40N4O14S
- Molecular Weight:580.60
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Storage:
Please store the product under the recommended conditions in the Certificate of Analysis.
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Biological Activity
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Chemical Information
- Purity & Documentation
- References
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Publications Citing Use of MedChemExpress (MCE) Exaluren sulfate
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Bio/Physico-chemical Assay
Biological Activity
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Cell Line
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Type | Value | Description | References |
|---|---|---|---|---|
| HeLa | IC50 |
1493 μM
Compound: (S)-9
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Inhibition of mitochondrial ribosome-mediated protein synthesis in human HeLa cells assessed as {35S]methionine incorporation by autoradiography
Inhibition of mitochondrial ribosome-mediated protein synthesis in human HeLa cells assessed as {35S]methionine incorporation by autoradiography
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[PMID: 23148581] |
Exaluren sulfate 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 sulfate 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 sulfate 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) sulfate 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.
| NCT Number | Sponsor | Condition | Start Date |
Phase
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|---|---|---|---|---|
| NCT01329991 | Plexxikon| | 2011-05 | PHASE1 |
Chemical Information
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CAS No. 1375073-94-1
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Molecular Weight 580.60
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Formula C19H40N4O14S
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SMILES
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.[x]
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Synonyms
ELX-02 sulfate; NB-124 sulfate
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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.
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 sulfate 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
Purity & Documentation
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]
Calculators
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)
Exaluren sulfate Related Classifications
HY-114231C Related Classifications
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