Viltolarsen| ChemScene
Viltolarsen (NS-065/NCNP-01), a phosphorodiamidate morpholino antisense oligonucleotide, targets the splicing of exon 53 in the dystrophin gene. Viltolarsen can be used for the research of the Duchenne muscular dystrophy (DMD).In Vitro: Duchenne muscular dystrophy (DMD) is one of the most common lethal muscle-wasting disorders affecting young boys caused by mutations in the DMD gene. Exon skipping has emerged as a promising therapy for DMD. Antisense oligonucleotides (AONs) are designed to induce the skipping of exon(s), in order to restore the reading frame, and therefore, allow for dystrophin expression.
Trivial name | Viltolarsen |
Catalog Number | CS-0204196 |
Alternative Name(s) | NS-065/NCNP-01 |
Molecular Formula | 6924.80 |
CAS# | 2055732-84-6 |
Purity | >98% |
Condensed Formula | C244H381N113O88P20 |
Size | 10mg |
Supplier Page | www.chemscene.com/2055732-84-6.html |