| Bioactivity | 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]. |
| Invitro | 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]. |
| Name | Casimersen |
| CAS | 1422958-19-7 |
| Molar Mass | 7584.00 |
| Appearance | Solid |
| Transport | Room temperature in continental US; may vary elsewhere. |
| Storage | -20°C, stored under nitrogen, away from moisture *In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture) |
| Reference | [1]. Shirley M. Casimersen: First Approval. Drugs. 2021;81(7):875-879. [2]. Casimersen. Am J Health Syst Pharm. 2021;78(13):1149-1150. |