| Chemical Properties | Back Directory | [storage temp. ]
Store at -20°C | [form ]
Solid | [color ]
White to off-white | [Sequence]
RNA, [P-deoxy-P-(dimethylamino)](2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a→5')(C-A-A-m5U-G-C-C-A-m5U-C-C-m5U-G-G-A-G-m5U-m5U-C-Cm5U-G), 5'-[P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N-dimethylphosphonamidate] |
| Hazard Information | Back Directory | [Uses]
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]. | [References]
[1] Shirley M. Casimersen: First Approval. Drugs. 2021;81(7):875-879. DOI:10.1007/s40265-021-01512-2
[2] Casimersen. Am J Health Syst Pharm. 2021;78(13):1149-1150. DOI:10.1093/ajhp/zxab138
[3] Carver MP, Charleston JS, Shanks C, et al. Toxicological Characterization of Exon Skipping Phosphorodiamidate Morpholino Oligomers (PMOs) in Non-human Primates. J Neuromuscul Dis. 2016;3(3):381-393. DOI:10.3233/JND-160157 |
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