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

Presence of phosphodiester backbone, but not nucleobases, in the guide's 3' terminal region is necessary for RISC loading and target cleavage in vitro and in vivo

引导RNA 3'末端区域中磷酸二酯骨架的存在(而非核碱基的存在)是RISC在体外和体内装载和靶标切割所必需的。

O'Reilly, Daniel; Furgal, Raymond C; Ross, Vella M; Luu, Eric; Gross, Katherine Y; Hariharan, Vignesh; Summers, Ashley; Cooper, David; Allen, Sarah; Dahlke, Christopher; Rachid, Mohamad Omar; Chan, Io Long; Fakih, Hassan H; Alterman, Julia F; Echeverria, Dimas; Watts, Jonathan K; Khvorova, Anastasia

Organyl 5'-Phosphates in siRNA Guide Strands: Structure-Function Relationships Governing Anchoring in Argonaute 2 and Metabolic Stability

siRNA引导链中的有机5'-磷酸酯:调控Argonaute 2锚定和代谢稳定性的结构-功能关系

Carrigan-Broda, Theodore; Gebert, Luca F R; Hildebrand, Samuel; Yamada, Nozomi; Luu, Eric; Caiazzi, Jillian; McHugh, Nicholas; Echeverria, Dimas; Wagh, Atish; Watts, Jonathan K; Khvorova, Anastasia; MacRae, Ian J; Yamada, Ken

Blocking somatic repeat expansion and lowering huntingtin via RNA interference synergize to prevent Huntington's disease pathogenesis in mice

通过RNA干扰抑制体细胞重复序列扩增和降低亨廷顿蛋白水平可协同预防小鼠亨廷顿病的发病机制。

Belgrad, Jillian; Summers, Ashley; Landles, Christian; Greene, Jonathan R; Hildebrand, Samuel; Knox, Emily; Sapp, Ellen; Yamada, Nozomi; Furgal, Raymond; Miller, Rachael; Osborne, Georgina F; Chase, Kathryn; Luu, Eric; Freedman, Jason; Bramato, Brianna; McHugh, Nicholas; Benoit, Vicky; O'Reilly, Daniel; Greer, Paul; Bates, Gillian P; Vogt, Thomas F; Lee, Ramee; Howland, David; DiFiglia, Marian; Aronin, Neil; Khvorova, Anastasia

A programmable dual-targeting siRNA scaffold supports potent two-gene modulation in the central nervous system.

可编程双靶向siRNA支架支持中枢神经系统中有效的双基因调控

Belgrad Jillian, Tang Qi, Hildebrand Sam, Summers Ashley, Sapp Ellen, Echeverria Dimas, O'Reilly Dan, Luu Eric, Bramato Brianna, Allen Sarah, Cooper David, Alterman Julia, Yamada Ken, Aronin Neil, DiFiglia Marian, Khvorova Anastasia

Di-valent siRNA-mediated silencing of MSH3 blocks somatic repeat expansion in mouse models of Huntington's disease

二价siRNA介导的MSH3沉默可阻断亨廷顿病小鼠模型中的体细胞重复序列扩增

O'Reilly, Daniel; Belgrad, Jillian; Ferguson, Chantal; Summers, Ashley; Sapp, Ellen; McHugh, Cassandra; Mathews, Ella; Boudi, Adel; Buchwald, Julianna; Ly, Socheata; Moreno, Dimas; Furgal, Raymond; Luu, Eric; Kennedy, Zachary; Hariharan, Vignesh; Monopoli, Kathryn; Yang, X William; Carroll, Jeffery; DiFiglia, Marian; Aronin, Neil; Khvorova, Anastasia

A programmable dual-targeting di-valent siRNA scaffold supports potent multi-gene modulation in the central nervous system.

可编程双靶向二价siRNA支架支持中枢神经系统中有效的多基因调控

Belgrad Jillian, Tang Qi, Hildebrand Sam, Summers Ashley, Sapp Ellen, Echeverria Dimas, O'Reilly Dan, Luu Eric, Bramato Brianna, Allen Sarah, Cooper David, Alterman Julia, Yamada Ken, Aronin Neil, DiFiglia Marian, Khvorova Anastasia