Casimersen (SRP-4045) is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer class that binds to exon 45 of dystrophin pre-mRNA, restores the open-reading frame through exon 45 skipping. Casimersen enables the production of an internally truncated yet functional dystrophin protein, thereby serving as a specialized molecular tool for studying exon-skipping mechanisms and therapeutic development in Duchenne muscular dystrophy research.
CAS Nummer:
[1422958-19-7]
Target-Kategorie:
Others
T38859
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