Viltolarsen

Viltolarsen (NS-065/NCNP-01) is a phosphorodiamidate morpholino antisense oligonucleotide. Viltolarsen 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 has the potential for Duchenne muscular dystrophy (DMD) research[1][2].

Product Specifications

CAS Number

[2055732-84-6]

Product Name Alternative

NS-065/NCNP-01

UNSPSC

12352208

Target

Dystrophin; Nucleoside Antimetabolite/Analog

Type

Oligonucleotides

Related Pathways

Cell Cycle/DNA Damage; Cytoskeleton

Field of Research

Metabolic Disease

Assay Protocol

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

Purity

95.25

Solubility

H2O : 100 mg/mL (ultrasonic)

Smiles

[Viltolarsen]

Molecular Formula

C244H381N113O88P20

Molecular Weight

6924.80

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.