Patisiran sodium 是一种双链小干扰 RNA，靶向 Transthyretin (TTR) 信使 RNA 内的序列。Patisiran sodium 特异性抑制突变体和野生型 TTR 的肝脏合成。Patisiran sodium 可用于研究遗传性 TTR 淀粉样蛋白症的研究。
货号:ajcx35546
CAS:1386913-72-9
分子式:N/A
分子量：N/A
溶解度:N/A
纯度：98%
存储：Store at -20°C
库存：现货

Background:

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 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].

[1]. Adams D, et, al. Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis. N Engl J Med. 2018 Jul 5;379(1):11-21.
[2]. Hoy SM. Patisiran: First Global Approval. Drugs. 2018 Oct;78(15):1625-1631.
[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.