PEG2000-C-DMG，一种脂质体，可用于制备 Onpattro。Onpattro 是一种肝脏导向的研究性 RNAi 治疗剂，通过靶向甲状腺素运载蛋白 mRNA的3′非翻译区，利用这一过程减少突变型和野生型甲状腺素运载蛋白的产生。
货号:ajcx36414
CAS:N/A
分子式:C38H73NO8
分子量：2706.38
溶解度:DMSO : 100 mg/mL (36.95 mM; Need ultrasonic)
纯度：98%
存储：Store at -20°C
库存：现货

Background:

PEG2000-C-DMG, a lipid, can be used for the preparation of Onpattro. Onpattro, a hepatically directed investigational RNAi therapeutic agent, harnesses this process to reduce the production of mutant and wild-type transthyretin by targeting the 3′ untranslated region of transthyretin mRNA[1][2].

[1]. Garber K. Alnylam launches era of RNAi drugs. Nat Biotechnol. 2018;36(9):777-778. [2]. Adams D, Gonzalez-Duarte A, O’Riordan WD, et al. Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis. N Engl J Med. 2018;379(1):11-21.