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

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

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

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

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

Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep

利用治疗性siRNA进行的给药途径比较研究表明,多塞特绵羊体内存在广泛的基因调控。

Ferguson, Chantal M; Godinho, Bruno Mdc; Alterman, Julia F; Coles, Andrew H; Hassler, Matthew; Echeverria, Dimas; Gilbert, James W; Knox, Emily G; Caiazzi, Jillian; Haraszti, Reka A; King, Robert M; Taghian, Toloo; Puri, Ajit; Moser, Richard P; Gounis, Matthew J; Aronin, Neil; Gray-Edwards, Heather; Khvorova, Anastasia

Diverse lipid conjugates for functional extra-hepatic siRNA delivery in vivo.

用于体内功能性肝外siRNA递送的多种脂质缀合物

Biscans Annabelle, Coles Andrew, Haraszti Reka, Echeverria Dimas, Hassler Matthew, Osborn Maire, Khvorova Anastasia

Serum Deprivation of Mesenchymal Stem Cells Improves Exosome Activity and Alters Lipid and Protein Composition

血清剥夺可改善间充质干细胞的外泌体活性并改变其脂质和蛋白质组成。

Haraszti, Reka Agnes; Miller, Rachael; Dubuke, Michelle L; Rockwell, Hannah E; Coles, Andrew H; Sapp, Ellen; Didiot, Marie-Cecile; Echeverria, Dimas; Stoppato, Matteo; Sere, Yves Y; Leszyk, John; Alterman, Julia F; Godinho, Bruno M D C; Hassler, Matthew R; McDaniel, Justice; Narain, Niven R; Wollacott, Rachel; Wang, Yang; Shaffer, Scott A; Kiebish, Michael A; DiFiglia, Marian; Aronin, Neil; Khvorova, Anastasia

Novel Cluster and Monomer-Based GalNAc Structures Induce Effective Uptake of siRNAs in Vitro and in Vivo

新型簇状和单体状 GalNAc 结构可诱导 siRNA 在体外和体内的有效吸收

Sharma, Vivek K; Osborn, Maire F; Hassler, Matthew R; Echeverria, Dimas; Ly, Socheata; Ulashchik, Egor A; Martynenko-Makaev, Yury V; Shmanai, Vadim V; Zatsepin, Timofei S; Khvorova, Anastasia; Watts, Jonathan K

Docosahexaenoic Acid Conjugation Enhances Distribution and Safety of siRNA upon Local Administration in Mouse Brain

二十二碳六烯酸偶联可增强siRNA在小鼠脑内局部给药后的分布和安全性

Nikan, Mehran; Osborn, Maire F; Coles, Andrew H; Godinho, Bruno Mdc; Hall, Lauren M; Haraszti, Reka A; Hassler, Matthew R; Echeverria, Dimas; Aronin, Neil; Khvorova, Anastasia