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

A translatable RNAi-driven gene therapy silences PMP22/Pmp22 genes and improves neuropathy in CMT1A mice

可翻译的 RNAi 驱动基因疗法可沉默 PMP22/Pmp22 基因并改善 CMT1A 小鼠的神经病变

Marina Stavrou, Alexia Kagiava, Sarah G Choudury, Matthew J Jennings, Lindsay M Wallace, Allison M Fowler, Amanda Heslegrave, Jan Richter, Christina Tryfonos, Christina Christodoulou, Henrik Zetterberg, Rita Horvath, Scott Q Harper, Kleopas A Kleopa

RNAi-Based Gene Therapy Rescues Developmental and Epileptic Encephalopathy in a Genetic Mouse Model

基于 RNAi 的基因疗法挽救了遗传小鼠模型中的发育性和癫痫性脑病

Osasumwen V Aimiuwu, Allison M Fowler, Megha Sah, Jia Jie Teoh, Ayla Kanber, Nettie K Pyne, Sabrina Petri, Chana Rosenthal-Weiss, Mu Yang, Scott Q Harper, Wayne N Frankel

Allele-specific RNA interference prevents neuropathy in Charcot-Marie-Tooth disease type 2D mouse models

等位基因特异性 RNA 干扰可预防 Charcot-Marie-Tooth 病 2D 型小鼠模型中的神经病变

Kathryn H Morelli, Laurie B Griffin, Nettie K Pyne, Lindsay M Wallace, Allison M Fowler, Stephanie N Oprescu, Ryuichi Takase, Na Wei, Rebecca Meyer-Schuman, Dattatreya Mellacheruvu, Jacob O Kitzman, Samuel G Kocen, Timothy J Hines, Emily L Spaulding, James R Lupski, Alexey Nesvizhskii, Pedro Mancias

AAV-mediated follistatin gene therapy improves functional outcomes in the TIC-DUX4 mouse model of FSHD

AAV 介导的卵泡抑素基因治疗可改善 TIC-DUX4 小鼠 FSHD 模型的功能结果

Carlee R Giesige, Lindsay M Wallace, Kristin N Heller, Jocelyn O Eidahl, Nizar Y Saad, Allison M Fowler, Nettie K Pyne, Mustafa Al-Kharsan, Afrooz Rashnonejad, Gholamhossein Amini Chermahini, Jacqueline S Domire, Diana Mukweyi, Sara E Garwick-Coppens, Susan M Guckes, K John McLaughlin, Kathrin Meyer

Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD

临床前安全性和脱靶研究,以支持 AAV 介导的 RNAi 疗法在 FSHD 中的应用

Lindsay M Wallace, Nizar Y Saad, Nettie K Pyne, Allison M Fowler, Jocelyn O Eidahl, Jacqueline S Domire, Danielle A Griffin, Adam C Herman, Zarife Sahenk, Louise R Rodino-Klapac, Scott Q Harper

Mouse Dux is myotoxic and shares partial functional homology with its human paralog DUX4

小鼠 Dux 具有肌毒性,与人类旁系同源物 DUX4 具有部分功能同源性

Jocelyn O Eidahl, Carlee R Giesige, Jacqueline S Domire, Lindsay M Wallace, Allison M Fowler, Susan M Guckes, Sara E Garwick-Coppens, Paul Labhart, Scott Q Harper