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

An siRNA targeting S6k1 identifies photoreceptor phospholipid metabolism as a contributor to lipid buildup in age-related macular degeneration.

针对 S6k1 的 siRNA 发现光感受器磷脂代谢是导致年龄相关性黄斑变性中脂质积累的因素之一。

期刊:Molecular Therapy-Nucleic Acids
影响因子:6.1
doi:10.1016/j.omtn.2026.102878
Cheng Shun-Yun, Giguere Delaney, Kim San, Seddon Johanna M, Caiazzi Jillian, Gross Katherine, McHugh Nicholas, Echeverria Dimas, Alterman Julia F, Gray-Edwards Heather, Benatti Hector Ribeiro, Renner Lauren, Woolard Hannah, Stoddard Jonathan, McGill Trevor J, Neuringer Martha, Brush Richard S, Agbaga Martin-Paul, Khvorova Anastasia, Punzo Claudio
代谢
S6k
脂代谢
发表日期：2026
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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

期刊:
影响因子:
doi:10.64898/2026.02.13.705631
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
信号转导
代谢
发表日期：2026
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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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A Therapeutic Small-Interfering RNA Potentiates Janus Kinase 1 Modulation for the Treatment of Dog Inflammatory Diseases.

治疗性小干扰RNA增强Janus激酶1调节作用，用于治疗犬类炎症性疾病

期刊:ACS Pharmacology and Translational Science
影响因子:3.7
doi:10.1021/acsptsci.4c00594
Tang Qi, Fakih Hassan H, Gross Katherine Y, Winter Sarah J, Bramato Brianna M, Yıldız-Altay Ümmügülsüm, Chang Benjamin K, Echeverria Dimas, Jackson Samuel O, McHugh Nicholas, Srnka Alyxandr, Alterman Julia F, Almela Ramón M, Kudej Raymond K, London Cheryl A, Richmond Jillian M, Harris John E, Khvorova Anastasia
免疫/内分泌
炎症
发表日期：2025
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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。

期刊:
影响因子:
doi:10.1101/2025.03.26.645328
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
MECP2
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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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

期刊:Molecular Therapy-Nucleic Acids
影响因子:6.1
doi:10.1016/j.omtn.2024.102291
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
发表日期：2024
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mRNA Nuclear Clustering Leads to a Difference in Mutant Huntingtin mRNA and Protein Silencing by siRNAs In Vivo.

mRNA 核聚集导致 siRNA 在体内对突变亨廷顿 mRNA 和蛋白质的沉默存在差异

期刊:Nucleic Acid Therapeutics
影响因子:4.7
doi:10.1089/nat.2024.0027
Allen Sarah, O'Reilly Daniel, Miller Rachael, Sapp Ellen, Summers Ashley, Paquette Joseph, Echeverria Moreno Dimas, Bramato Brianna, McHugh Nicholas, Yamada Ken, Aronin Neil, DiFiglia Marian, Khvorova Anastasia
免疫/内分泌
发表日期：2024
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Preventing acute neurotoxicity of CNS therapeutic oligonucleotides with the addition of Ca(2+) and Mg(2+) in the formulation

在制剂中添加Ca(2+)和Mg(2+)可预防中枢神经系统治疗性寡核苷酸的急性神经毒性

期刊:
影响因子:
doi:10.1101/2024.06.06.597639
Miller, Rachael; Paquette, Joseph; Barker, Alexandra; Sapp, Ellen; McHugh, Nicholas; Bramato, Brianna; Yamada, Nozomi; Alterman, Julia; Echeveria, Dimas; Yamada, Ken; Watts, Jonathan; Anaclet, Christelle; DiFiglia, Marian; Khvorova, Anastasia; Aronin, Neil
毒理研究
神经科学
发表日期：2024
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