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

Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington's disease models

治疗性 siRNA 的化学工程用于亨廷顿氏病模型中的等位基因特异性基因沉默

Faith Conroy #, Rachael Miller #, Julia F Alterman, Matthew R Hassler, Dimas Echeverria, Bruno M D C Godinho, Emily G Knox, Ellen Sapp, Jaquelyn Sousa, Ken Yamada, Farah Mahmood, Adel Boudi, Kimberly Kegel-Gleason, Marian DiFiglia, Neil Aronin, Anastasia Khvorova, Edith L Pfister

Structurally constrained phosphonate internucleotide linkage impacts oligonucleotide-enzyme interaction, and modulates siRNA activity and allele specificity

结构受限的膦酸酯核苷酸间键影响寡核苷酸-酶相互作用,并调节 siRNA 活性和等位基因特异性

Ken Yamada, Samuel Hildebrand, Sarah M Davis, Rachael Miller, Faith Conroy, Ellen Sapp, Jillian Caiazzi, Julia F Alterman, Loic Roux, Dimas Echeverria, Matthew R Hassler, Edith L Pfister, Marian DiFiglia, Neil Aronin, Anastasia Khvorova

Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease

人工 miRNA 可降低亨廷顿氏病转基因绵羊模型中纹状体中的人类突变型亨廷顿蛋白

Edith L Pfister, Natalie DiNardo, Erica Mondo, Florie Borel, Faith Conroy, Cara Fraser, Gwladys Gernoux, Xin Han, Danjing Hu, Emily Johnson, Lori Kennington, PengPeng Liu, Suzanne J Reid, Ellen Sapp, Petr Vodicka, Tim Kuchel, A Jennifer Morton, David Howland, Richard Moser, Miguel Sena-Esteves, Guan

Safe and Efficient Silencing with a Pol II, but Not a Pol lII, Promoter Expressing an Artificial miRNA Targeting Human Huntingtin

使用 Pol II 而非 Pol lII 启动子进行安全有效的沉默,表达针对人类亨廷顿蛋白的人工 miRNA

Edith L Pfister, Kathryn O Chase, Huaming Sun, Lori A Kennington, Faith Conroy, Emily Johnson, Rachael Miller, Florie Borel, Neil Aronin, Christian Mueller

HTT-lowering reverses Huntington's disease immune dysfunction caused by NFκB pathway dysregulation

降低 HTT 可逆转 NFκB 通路失调引起的亨廷顿病免疫功能障碍

Ulrike Träger, Ralph Andre, Nayana Lahiri, Anna Magnusson-Lind, Andreas Weiss, Stephan Grueninger, Chris McKinnon, Eva Sirinathsinghji, Shira Kahlon, Edith L Pfister, Roger Moser, Holger Hummerich, Michael Antoniou, Gillian P Bates, Ruth Luthi-Carter, Mark W Lowdell, Maria Björkqvist, Gary R Ostroff