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

Intradermal delivery of lipophilic siRNAs enables prolonged skin retention and sustained gene silencing in a porcine model.

在猪模型中，皮内注射亲脂性siRNA可实现皮肤长期滞留和持续基因沉默。

期刊:Nature Communications
影响因子:15.7
doi:10.1038/s41467-026-68993-1
Fakih Hassan H, Zain Ui Abideen Mohammad, Rachid Mohamad Omar, Gross Katherine Y, Ormsby Thomas J R, Ross Vella M, Gagnon Rosemary, Dahlke Christopher, Furgal Raymond C, Lochmann Clemens, Summers Ashley, Sleiman Hanadi F, Fürst Sylvia M, Jackson Shanté N, Gordilho Juliana O, Bouix-Peter Claire, Portal Thibaud, Tang Qi, Harris John E, Khvorova Anastasia, Blanchard Carine, Alterman Julia F
表观遗传
发表日期：2026
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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。

期刊:Nucleic Acids Research
影响因子:13.1
doi:10.1093/nar/gkaf1438
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
发表日期：2026
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Potent and durable gene modulation in heart and muscle with chemically defined lipophilic siRNAs

利用化学成分明确的亲脂性siRNA在心脏和肌肉中实现高效持久的基因调控

期刊:Nucleic Acids Research
影响因子:13.1
doi:10.1093/nar/gkaf1374
Fakih, Hassan H; Lochmann, Clemens; Gagnon, Rosemary; Summers, Ashley; Caiazzi, Jillian; Buchwald, Julianna E; Tang, Qi; Maru, Bruktawit; Hildebrand, Samuel R; Abideen, Mohammad Zain U I; Furgal, Raymond C; Gross, Katherine Y; Yang, Yeon-Suk; Cooper, David; Monopoli, Kathryn R; Echeverria, Dimas; Shim, Jae-Hyuck; Yamada, Ken; Alterman, Julia F; Khvorova, Anastasia
信号转导
发表日期：2025
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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

期刊:Nucleic Acids Research
影响因子:13.1
doi:10.1093/nar/gkaf479
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
其它
发表日期：2025
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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。

期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2025.05.010
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
表观遗传
SOD1
ALS
发表日期：2025
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Systematic Evaluation of Position-Specific Tolerability of Seven Backbone and Ribose Modifications in Fully Chemically Stabilized siRNAs

对七种骨架和核糖修饰在完全化学稳定的siRNA中的位置特异性耐受性进行系统评价

期刊:Nucleic Acid Therapeutics
影响因子:4.7
doi:10.1089/nat.2024.0077
O'Reilly, Daniel; Furgal, Raymond; Hariharan, Vignesh; Lochmann, Clemens; Cooper, David; Echeverria, Dimas; Khvorova, Anastasia
发表日期：2025
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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。

期刊:
影响因子:
doi:10.1101/2025.06.24.661398
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
细胞生物学
发表日期：2025
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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

期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2023.09.016
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
细胞生物学
多发性硬化症
发表日期：2023
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