1375073-94-1
Chemical Structure
Exaluren sulfate
Synonym(s): ELX-02 sulfate; NB-124 sulfate
- CAS No.: 1375073-94-1
- Formula:C19H40N4O14S
- Molecular Weight:580.60
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]
Biological Activity: 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[1][2].
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Exaluren sulfate | 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. | |||||||||||||||||||||
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- [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]
Keywords
