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。

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

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

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

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

A systemically deliverable lipid-conjugated siRNA targeting DUX4 as an facioscapulohumeral muscular dystrophy therapeutic.

一种可系统递送的脂质偶联 siRNA,靶向 DUX4,作为面肩肱型肌营养不良症的治疗方法

Daman Katelyn, Yan Jing, Biscans Annabelle, Echeverria Dimas, Shmushkovich Taisia, Wolfson Alexey, Alterman Julia F, Khvorova Anastasia, Emerson Charles P Jr

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

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

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