Casimersen (SRP-4045) 是磷酸二酰胺吗啉寡聚物亚类的反义寡核苷酸。Casimersen 与肌营养不良蛋白前体 mRNA 的外显子 45 结合，恢复开放阅读框 (通过跳过外显子45)，从而产生一种内部截短但功能性的肌营养不良蛋白。Casimersen 可用于杜氏肌营养不良症 (DMD) 的研究。
货号:ajcx37466
CAS:1422958-19-7
分子式:N/A
分子量：N/A
溶解度:N/A
纯度：98%
存储：Store at -20°C
库存：现货

Background:

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].

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].

[1]. Shirley M. Casimersen: First Approval. Drugs. 2021;81(7):875-879. [2]. Casimersen. Am J Health Syst Pharm. 2021;78(13):1149-1150.