日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

Systematic analysis of siRNA and mRNA features impacting fully chemically modified siRNA efficacy.

对影响完全化学修饰的siRNA功效的siRNA和mRNA特征进行系统分析

Davis Sarah M, Hildebrand Samuel, MacMillan Hannah J, Monopoli Kathryn R, Buchwald Julianna, Sousa Jacquelyn, Cooper David, Ly Socheata, Echeverria Dimas, McHugh Nicholas, Ferguson Chantal, Coles Andrew, Hariharan Vignesh N, O'Reilly Daniel, Tang Qi, Furgal Raymond, Yamada Ken, Alterman Julia F, Gilbert James W, Knox Emily, Pineda Yamilett, Weston Caitlyn N, Baer Christina E, Pai Athma A, Khvorova Anastasia

Single-dose administration of therapeutic divalent siRNA targeting MECP2 prevents lethality for one year in an MECP2 duplication mouse model

单次注射靶向 MECP2 的治疗性二价 siRNA 可使 MECP2 重复小鼠模型免于死亡一年。

Hariharan, Vignesh N; Summers, Ashley; Clipperton-Allen, Amy E; Caiazzi, Jillian; Hildebrand, Samuel R; O'Reilly, Daniel; Tang, Qi; Kennedy, Zachary; Echeverria, Dimas; McHugh, Nicholas; Cooper, David; Souza, Jacqueline; Ferguson, Chantal; Bogdanik, Laurent; Coenraads, Monica; Khvorova, Anastasia

Phosphatidylcholine head group chemistry alters the extrahepatic accumulation of lipid-conjugated siRNA

磷脂酰胆碱头部基团的化学性质会改变脂质偶联siRNA在肝外的积累。

Hariharan, Vignesh N; Nakamura, Takahiro; Shin, Minwook; Tang, Qi; Sontakke, Vyankat; Caiazzi, Jillian; Hildebrand, Samuel; Khvorova, Anastasia; Yamada, Ken