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.
Publications Citing Use of MedChemExpress (MCE) Exaluren sulfate
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Bio/Physico-chemical Assay
Biological Activity
Description
Cellular Effect
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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] |
In Vitro
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. Further protocols information, click here.
In Vivo
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.
Clinical Trial
| 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
Protocols
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Fibrosis/Collagen Morphometry
Fibrosis and collagen morphometry is based on the quantitative visualization of fibrillar collagen deposition in tissue sections using histochemical stains such as Sirius Red (Picrosirius Red) or Masson's trichrome, followed by image-based or polarization-enhanced analysis to estimate collagen proportional area as a surrogate of extracellular matrix accumulation during fibrotic remodeling. Sirius Red combined with polarized light microscopy enhances detection of collagen fibers due to birefringence properties, enabling more specific visualization of collagen type I and III fibrils compared to conventional bright-field histology, while whole-section or region-restricted digital morphometry reduces field-selection bias in fibrosis assessment. Alternative quantitative approaches include second harmonic generation (SHG) and two-photon excited fluorescence microscopy, which enable label-free detection of fibrillar collagen and have been validated against histological staining and biochemica
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Collagen: Sirius Red Staining
Sirius Red or picrosirius red staining is a histochemical method for visualizing collagen-rich extracellular matrix in tissue sections, and collagen fibers are detected as red-stained structures under bright-field microscopy with enhanced birefringence under polarized light. Picrosirius red is useful for assessing total collagen organization, distribution, and fibrosis burden, but polarized color should not be interpreted as a definitive collagen type I versus type III readout because color is affected by fiber orientation, thickness, and packing.
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Connective Tissue: Masson's Trichrome/Collagen Trichrome Staining
Masson’s Trichrome (collagen/trichrome staining) is a histological technique that differentially stains tissue compartments using sequential acidic dyes to distinguish collagen from muscle and cytoplasmic components based on dye affinity and tissue permeability differences, enabling visualization of fibrosis and connective tissue architecture in histological sections. The classical formulation typically uses Weigert's iron hematoxylin for nuclear staining, Biebrich scarlet-acid fuchsin for cytoplasm and muscle, and aniline blue (or light green variants) for collagen, producing a characteristic blue/green collagen signal contrasted against red cytoplasm and dark nuclei. The staining principle relies on selective displacement of smaller dye molecules by larger anionic dyes in collagen-rich regions under controlled acidified conditions, which enhances collagen-specific dye retention. This property makes the method widely used for fibrosis assessment in organs such as heart, liver, lung, a
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)