Viltolarsen sodium
Based on 1 Customer Validation
Viltolarsen (NS-065/NCNP-01) sodium is a phosphorodiamidate morpholino antisense oligonucleotide. Viltolarsen sodium binds to exon 53 of the dystrophin mRNA precursor and restores the amino acid open-reading frame by skipping exon 53, resulting in the production of a shortened dystrophin protein that contains essential functional portions. Viltolarsen sodium has the potential for Duchenne muscular dystrophy (DMD) research.
For research use only. We do not sell to patients.
- Purity: 95.25%
- Formula: C244H360N113Na21O88P20
- Molecular Weight:6924.8 (free acid)
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Storage:
-20°C, stored under nitrogen, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture)
Biological Activity
Viltolarsen (NS-065/NCNP-01; 2 days) sodium induces exon 53 skipping in cells from a DMD model with a deletion of exons 45-52 with an EC50 value of 0.63 μM and in cells with a deletion of exons 48-52 with an EC50 value of 2.3 μM[2].
Viltolarsen (10 μM; 3 days) sodium causes the dystrophin protein expression in cells from a DMD model[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 6924.8 (free acid)
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Formula C244H360N113Na21O88P20
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Color White to off-white
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SMILES
[Viltolarsen (sodium)]
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Synonyms
NS-065/NCNP-01 sodium
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Shipping
Room temperature in continental US; may vary elsewhere.
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Storage
-20°C, stored under nitrogen, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture)
Purity & Documentation
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Data Sheet (266 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
[1]. Sohita Dhillon. Viltolarsen: First Approval. Drugs. 2020 Jul;80(10):1027-1031. [Content Brief]
[2]. Naoki Watanabe, et al. NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy. Mol Ther Nucleic Acids. 2018 Dec 7:13:442-449. [Content Brief]
Calculators
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)