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

RNAi-mediated silencing of SOD1 profoundly extends survival and functional outcomes in ALS mice

RNAi介导的SOD1基因沉默显著延长了ALS小鼠的生存期并改善了其功能预后。

Weiss, Alexandra; Gilbert, James W; Rivera Flores, Iris Valeria; Belgrad, Jillian; Ferguson, Chantal; Dogan, Elif O; Wightman, Nicholas; Mocarski, Kit; Echeverria, Dimas; Harkins, Ashley L; Summers, Ashley; Bramato, Brianna; McHugh, Nicholas; Furgal, Raymond; Yamada, Nozomi; Cooper, David; Monopoli, Kathryn; Godinho, Bruno M D C; Hassler, Matthew R; Yamada, Ken; Greer, Paul; Henninger, Nils; Brown, Robert H Jr; Khvorova, Anastasia

Identification of selective and non-selective C9ORF72 targeting in vivo active siRNAs

鉴定选择性和非选择性靶向C9ORF72的体内活性siRNA

Gilbert, James W; Kennedy, Zachary; Godinho, Bruno M D C; Summers, Ashley; Weiss, Alexandra; Echeverria, Dimas; Bramato, Brianna; McHugh, Nicholas; Cooper, David; Yamada, Ken; Hassler, Matthew; Tran, Hélène; Gao, Fen Biao; Brown, Robert H Jr; Khvorova, Anastasia

Intrathecal Delivery of Therapeutic Oligonucleotides for Potent Modulation of Gene Expression in the Central Nervous System

鞘内注射治疗性寡核苷酸以强效调节中枢神经系统基因表达

Kennedy, Zachary; Gilbert, James W; Godinho, Bruno M D C

Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep

利用治疗性siRNA进行的给药途径比较研究表明,多塞特绵羊体内存在广泛的基因调控。

Ferguson, Chantal M; Godinho, Bruno Mdc; Alterman, Julia F; Coles, Andrew H; Hassler, Matthew; Echeverria, Dimas; Gilbert, James W; Knox, Emily G; Caiazzi, Jillian; Haraszti, Reka A; King, Robert M; Taghian, Toloo; Puri, Ajit; Moser, Richard P; Gounis, Matthew J; Aronin, Neil; Gray-Edwards, Heather; Khvorova, Anastasia