Casimersen

Names

[ Name ]:
Casimersen

Biological Activity

[Description]:

Casimersen (SRP-4045) is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer subclass. Casimersen binds to exon 45 of dystrophin pre-mRNA, restores the open-reading frame (by skipping exon 45) resulting in the production of an internally truncated but functional dystrophin protein. Casimersen can be used for the research of Duchenne muscular dystrophy (DMD)[1].

[Related Catalog]:

Research Areas >> Others

Signaling Pathways >> Others >> Others

[In Vitro]

Exon 45 skipping is intended to allow for production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 45 skipping[2].

[References]

[1]. Shirley M. Casimersen: First Approval. Drugs. 2021;81(7):875-879.

[2]. Casimersen. Am J Health Syst Pharm. 2021;78(13):1149-1150.

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.