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别名 帕替西兰钠
Patisiran sodium 是一种双链小干扰 RNA(siRNA),可选择性结合转甲状腺素蛋白(TTR)信使 RNA 中的特定序列,对肝脏中野生型和突变型 TTR 蛋白的合成产生强效而特异的抑制作用。Patisiran sodium 基于机制的活性奠定了其在遗传性 TTR 淀粉样变性研究、基因沉默治疗策略开发及 RNA 干扰转化医学中的重要应用。

Patisiran sodium 是一种双链小干扰 RNA(siRNA),可选择性结合转甲状腺素蛋白(TTR)信使 RNA 中的特定序列,对肝脏中野生型和突变型 TTR 蛋白的合成产生强效而特异的抑制作用。Patisiran sodium 基于机制的活性奠定了其在遗传性 TTR 淀粉样变性研究、基因沉默治疗策略开发及 RNA 干扰转化医学中的重要应用。
| 规格 | 价格 | 库存 | 数量 |
|---|---|---|---|
| 1 mg | ¥ 2,750 | 2-4周 | |
| 5 mg | ¥ 6,050 | 2-4周 | |
| 10 mg | ¥ 9,100 | 2-4周 |
| 产品描述 | Patisiran sodium is a double-stranded small interfering RNA (siRNA) that selectively binds a defined sequence within transthyretin (TTR) messenger RNA. Patisiran sodium possess potent and specific suppression of hepatic synthesis of both mutant and wild-type TTR protein, and its mechanism-based activity underpins its significant application in hereditary TTR amyloidosis research, gene silencing therapeutics development, and RNA interference–based translational medicine. |
| 体外活性 | Patisiran sodium causes TTR degradation (via RNA interference) and subsequently a reduction in serum TTR protein levels and tissue TTR protein deposits by specifically binding to a genetically conserved sequence in the 3' untranslated region of mutant and wild-type transthyretin (TTR) messenger RNA[2]. |
| 别名 | 帕替西兰钠 |
| CAS No. | 1386913-72-9 |
| Sequence | RNA, (A-U-G-G-A-A-Um-A-C-U-C-U-U-G-G-U-Um-A-C-dT-dT), complex with RNA (G-Um-A-ACm-Cm-A-A-G-A-G-Um-A-Um-Um-Cm-Cm-A-Um-dT-dT) (1:1) sodium salt |
| 存储 | In solvent: -80°C for 1 year | Shipping with blue ice/Shipping at ambient temperature. |
| 溶解度信息 | H2O: 25 mg/mL, Sonication is recommended. |
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