PEG2000-C-DMG, a pegylated 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.
性状
Solid
运输条件
Room temperature in continental US; may vary elsewhere.
储存方式
-20°C, stored under nitrogen In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen)
参考文献
[1]. Garber K. Alnylam launches era of RNAi drugs. Nat Biotechnol. 2018;36(9):777-778. [2]. Adams D, Gonzalez-Duarte A, ORiordan WD, et al. Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis. N Engl J Med. 2018;379(1):11-21.
溶解度数据
In Vitro: DMSO : 100 mg/mL (38.31 mM; Need ultrasonic)配制储备液