Viltolarsen

Names

[ CAS No. ]:
2055732-84-6

[ Name ]:
Viltolarsen

Biological Activity

[Description]:

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)[1].

[Related Catalog]:

Research Areas >> Others

Signaling Pathways >> Others >> Others

[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[1].

[References]

[1]. Dzierlega K, et al. Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy. Gene Ther. 2020;27(9):407-416.

Chemical & Physical Properties

No Any Chemical & Physical Properties

Related Compounds

The content on this webpage is sourced from various professional data sources. If you have any questions or concerns regarding the content, please feel free to contact service1@chemsrc.com.