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

Divalent siRNA for prion disease

用于朊病毒病的二价siRNA

Gentile, Juliana E; Corridon, Taylor L; Serack, Fiona E; Echeverria, Dimas; Kennedy, Zachary C; Gallant-Behm, Corrie L; Hassler, Matthew R; Kinberger, Garth A; Kelemen, Margaret N; Kamath, Nikita G; Lian, Yuan; Gross, Katherine Y; Miller, Rachael; DeSouza-Lenz, Kendrick; Howard, Michael; Guzman, Kenia; Chan, Nathan; Laversenne, Vanessa; Curtis, Daniel; Fettes, Kevin; Lemaitre, Marc; Jackson, Aimee L; Yamada, Ken; Alterman, Julia F; Coffey, Alissa A; Minikel, Eric Vallabh; Khvorova, Anastasia; Vallabh, Sonia M

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

Therapeutic delivery of albumin-binding siRNA targeting IRS2 to diverse cell types reduces mammary tumor growth.

将靶向 IRS2 的白蛋白结合 siRNA 治疗性递送至多种细胞类型可减少乳腺肿瘤的生长。

Tocheny Claire E, Buchwald Julianna E, Dahlke Christopher D, Fakih Hassan H, Morgan Jennifer S, Summers Ashley, Silva Christi A, Jackson Samuel O, Lee Ji-Sun, Card Michael-Anthony, Echeverria Dimas, Peterson Cornelia, Mercurio Arthur M, Khvorova Anastasia, Shaw Leslie M

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。

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

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

Divalent siRNA for prion disease.

用于朊病毒病的二价siRNA。

Gentile Juliana E, Corridon Taylor L, Serack Fiona E, Echeverria Dimas, Kennedy Zachary C, Gallant-Behm Corrie L, Hassler Matthew R, Kinberger Garth A, Kelemen Margaret N, Kamath Nikita G, Lian Yuan, Gross Katherine Y, Miller Rachael, DeSouza-Lenz Kendrick, Howard Michael, Guzman Kenia, Chan Nathan, Laversenne Vanessa, Curtis Daniel T, Fettes Kevin, Lemaitre Marc, Jackson Aimee L, Yamada Ken, Alterman Julia F, Coffey Alissa A, Minikel Eric Vallabh, Khvorova Anastasia, Vallabh Sonia M

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

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

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

Optimized chemical structure extends silencing duration of therapeutic siRNAs in dividing cancer and immune cells

优化的化学结构延长了治疗性siRNA在分裂的癌细胞和免疫细胞中的沉默持续时间。

Kremer, Anastasia; Ryaykenen, Tatyana; Segarra-Visent, Xavier; Sauer, Melanie; Breuer, Leon; Wu, Yiying; Tang, Qi; Cooper, David A; Echeverria, Dimas; Philouze, Clothilde; Bayon, Emilie; Georgess, Dan; Haraszti, Reka A