Ultevursen sodium
Based on 1 Customer Validation
Ultevursen sodium (QR-421a) is a splice-modulating antisense oligonucleotide targeting exon 13 of the USH2A gene, which restores the functional expression of Usherin protein by inducing exon skipping. Ultevursen sodium binds to USH2A pre-mRNA and modulates the splicing process to specifically skip exon 13 carrying the pathogenic mutation c.2299delG, generating an in-frame transcript and a truncated yet functionally normal protein. Ultevursen sodium exhibits concentration-dependent exon skipping activity in human cells and retinal organoid models, and restores Usherin expression and retinal function in zebrafish and gene-edited mouse models. Ultevursen sodium can be used for related research on type 2 Usher syndrome and non-syndromic retinitis pigmentosa.
For research use only. We do not sell to patients.
- Purity : 94.24%
- Formula: C273H370N81Na21O144P20S20
- Molecular Weight:8372.24 (free acid)
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Storage:
-20°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
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
- Protocols
- Purity & Documentation
- References
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Help & FAQs
Biological Activity
Description
In Vitro
Ultevursen sodium induces concentration-dependent USH2A exon 13 skipping in WERI-Rb1 cells[1].
Ultevursen sodium induces concentration-dependent USH2A exon 13 skipping in patient-derived iPSC-derived photoreceptor progenitor cells[1].
Ultevursen (QR-421a) (50 ng; 3 days) sodium delivered as a U7 snRNA skipper induces USH2A exon 13 skipping in stable HEK293T USH2A-eGFP reporter cells, leading to detectable eGFP expression[2].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only. Further protocols information, click here.
In Vivo
Ultevursenassociated AAV-U7 dual skipper (1 μL viral vector suspension; intratympanic + utricle microinjection; P1-P2 neonatal Ush2a KO-1/KO-1 mice; 16 weeks in vivo incubation period) sodium achieves robust hair cell transduction and mediates sustained Ush2a exon skipping within mouse inner ear tissue[2].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
Chemical Information
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Appearance Solid
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Molecular Weight 8372.24 (free acid)
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Formula C273H370N81Na21O144P20S20
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Color White to off-white
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SMILES
[Ultevursen (sodium)]
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Synonyms
QR-421a sodium
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Shipping
Room temperature in continental US; may vary elsewhere.
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Storage
-20°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
Protocols
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RNA extraction experimental
By lysing cells, releasing RNA, and removing impurities such as proteins and DNA, high-purity RNA products are finally obtained. The commonly used traditional method is the guanidine isothiocyanate/phenol/chloroform method (Trizol), which is suitable for a variety of animal materials including animal tissues, microorganisms, cultured cells, etc., and most plant materials.
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Genotoxicity/Mutagenicity Study
The bacterial reverse mutation assay detects point mutations that restore amino-acid prototrophy in auxotrophic Salmonella typhimurium or Escherichia coli tester strains; after exposure to a test article, mutagenic activity is read out as an increased number of revertant colonies on minimal agar compared with the vehicle control. The assay uses tester strains with different mutation targets so that base-substitution and frameshift mutagens can be detected, and testing is performed with and without exogenous mammalian metabolic activation because some chemicals require biotransformation to become mutagenic.
Purity & Documentation
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Data Sheet (267 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 (2242 KB)
References
Calculators
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)
Ultevursen sodium Related Classifications
HY-147412A Related Classifications
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