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

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

Suppression of Huntington's Disease Somatic Instability by Transcriptional Repression and Direct CAG Repeat Binding.

通过转录抑制和直接 CAG 重复序列结合抑制亨廷顿病体细胞不稳定性。

Mathews Ella W, Coffey Sydney R, Gärtner Annette, Belgrad Jillian, Bragg Robert M, O'Reilly Daniel, Cantle Jeffrey P, McHugh Cassandra, Summers Ashley, Fentz Joachim, Schwagarus Tom, Cornelius Antje, Lingos Ioannis, Burch Zoe, Kovalenko Marina, Andrew Marissa A, Bennett C Frank, Kordasiewicz Holly B, Marchionini Deanna M, Wilkinson Hilary, Vogt Thomas F, Beuzer Paolo, Pinto Ricardo M, Khvorova Anastasia, Howland David, Wheeler Vanessa C, Carroll Jeffrey B

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

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

Albumin-binding dendrimer-conjugated siRNA enables safe and effective gene silencing throughout the central nervous system

白蛋白结合树状聚合物偶联的siRNA能够安全有效地在整个中枢神经系统中进行基因沉默。

Fakih, Hassan H; Ohara, Masahiro; Summers, Ashley; Sarli, Samantha L; Kelly, Karen; Gagnon, Rosemary; Maru, Bruktawit; Bramato, Brianna; Khvorova, Anastasia; Watts, Jonathan K

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。

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

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; Wisniewski, Christi A; Jackson, Samuel O; Lee, Ji-Sun; Card, Michael-Anthony; Echeverria, Dimas; Peterson, Cornelia; Mercurio, Arthur M; Khvorova, Anastasia; Shaw, Leslie M

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。

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