Patisiran sodium 

Patisiran sodium is a double-stranded small interfering RNA that targets a sequence within the transthyretin (TTR) messenger RNA. Patisiran sodium specifically inhibits hepatic synthesis of mutant and wild-type TTR. Patisiran sodium can be used for the research of hereditary TTR amyloidosis^[1][2][3].

Patisiran sodium 导致 TTR 降解 (通过 RNA 干扰)，随后通过特异性结合突变体和野生型转甲状腺素蛋白 (TTR) 信使 RNA 的 3' 非翻译区中的遗传保守序列，降低血清 TTR 蛋白水平和组织 TTR 蛋白沉积^[2]。

MCE has not independently confirmed the accuracy of these methods. They are for reference only.

13427.10 (AS: 6661.3; SS: 6765.8)

1386913-72-9

固体

Off-white to light yellow

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

Room temperature in continental US; may vary elsewhere.

-20°C, stored under nitrogen

*In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen)

• [1]. Adams D, et, al. Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis. N Engl J Med. 2018 Jul 5;379(1):11-21.  [Content Brief]

  [2]. Hoy SM. Patisiran: First Global Approval. Drugs. 2018 Oct;78(15):1625-1631.  [Content Brief]

  [3]. Kristen AV, et, al. Patisiran, an RNAi therapeutic for the treatment of hereditary transthyretin-mediated amyloidosis. Neurodegener Dis Manag. 2019 Feb;9(1):5-23.  [Content Brief]