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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.
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.
规格 | 价格 | 库存 | 数量 |
---|---|---|---|
1 mg | ¥ 7,660 | 期货 | |
5 mg | ¥ 19,900 | 期货 | |
10 mg | ¥ 29,900 | 期货 |
产品描述 | 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. |
体外活性 | 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]. |
别名 | SRP-4045 |
CAS No. | 1422958-19-7 |
密度 | no data available |
存储 | Powder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice. |
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