Viltolarsen (sodium)

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[1][2].

Product Specifications

Product Name Alternative

NS-065/NCNP-01 (sodium)

UNSPSC

12352208

Target

Dystrophin; Nucleoside Antimetabolite/Analog

Type

Oligonucleotides

Related Pathways

Cell Cycle/DNA Damage; Cytoskeleton

Applications

Metabolism-protein/nucleotide metabolism

Field of Research

Metabolic Disease

Assay Protocol

https://www.medchemexpress.com/viltolarsen-sodium.html

Purity

95.25

Solubility

10 mM in H2O

Smiles

[Viltolarsen(sodium)]

Molecular Formula

C244H360N113Na21O88P20

Molecular Weight

6924.8 (free acid)

References & Citations

[1]Sohita Dhillon. Viltolarsen: First Approval. Drugs. 2020 Jul;80 (10) :1027-1031. |[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.