Powder: -20°C for 3 years | In solvent: -80°C for 1 year
Casimersen (SRP-4045) is an antisense oligonucleotide, specifically belonging to the phosphorodiamidate morpholino oligomer subclass. It binds to exon 45 of dystrophin pre-mRNA, effectively restoring the open-reading frame by skipping exon 45. This action ultimately leads to the production of a functional dystrophin protein, albeit internally truncated. Casimersen finds utility in Duchenne muscular dystrophy (DMD) research.
説明 | Casimersen (SRP-4045) is an antisense oligonucleotide, specifically belonging to the phosphorodiamidate morpholino oligomer subclass. It binds to exon 45 of dystrophin pre-mRNA, effectively restoring the open-reading frame by skipping exon 45. This action ultimately leads to the production of a functional dystrophin protein, albeit internally truncated. Casimersen finds utility in Duchenne muscular dystrophy (DMD) research. |
In vitro | Exon 45 skipping aims to facilitate the production of a shorter, internally truncated form of dystrophin protein in patients whose genetic mutations make them suitable candidates for Exon 45 skipping[2]. |
別名 | SRP-4045 |
分子量 | N/A |
CAS No. | 1422958-19-7 |
Powder: -20°C for 3 years | In solvent: -80°C for 1 year
You can also refer to dose conversion for different animals. 詳細
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Casimersen 1422958-19-7 SRP 4045 SRP-4045 SRP4045 Inhibitor inhibitor inhibit