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Golodirsen (SRP-4053) is a phosphorodiamidate morpholino oligomer (PMO) designed to selectively bind and target exon 53 of dystrophin pre-mRNA, with applications in the investigation of Duchenne muscular dystrophy (DMD).
Golodirsen (SRP-4053) is a phosphorodiamidate morpholino oligomer (PMO) designed to selectively bind and target exon 53 of dystrophin pre-mRNA, with applications in the investigation of Duchenne muscular dystrophy (DMD).
规格 | 价格 | 库存 | 数量 |
---|---|---|---|
25 mg | ¥ 10,600 | 期货 |
产品描述 | Golodirsen (SRP-4053) is a phosphorodiamidate morpholino oligomer (PMO) designed to selectively bind and target exon 53 of dystrophin pre-mRNA, with applications in the investigation of Duchenne muscular dystrophy (DMD). |
体外活性 | Golodirsen specifically targets exon 53 of dystrophin pre-mRNA, resulting in its exclusion from the final mRNA product. Skipping of exon 53 results in restoration of the mRNA reading frame and leads to the production of a partially internally deleted dystrophin protein with intact C and N-terminal regions[2]. |
别名 | SRP-4053, Golodirsen |
CAS No. | 1422959-91-8 |
存储 | Powder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice. |
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