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

Reduction of RAD23A extends lifespan and mitigates pathology in a mouse model of TDP-43 proteinopathy.

RAD23A 的减少可延长 TDP-43 蛋白病小鼠模型的寿命并减轻病理。

Guo Xueshui, Prajapati Ravindra Singh, Chun Jiyeon, Byun Insuk, Gebis Kamil K, Wang Yi-Zhi, Ling Karen, Dalton Casey, Blair Jeff A, Hamidianjahromi Anahid, Bachmann Gemma, Rigo Frank, Jafar-Nejad Paymaan, Savas Jeffrey N, Lee Min Jae, Sreedharan Jemeen, Kalb Robert G

Acute neuronal inhibition response caused by phosphorothioate antisense oligonucleotides following local delivery to the central nervous system

局部递送硫代磷酸酯反义寡核苷酸至中枢神经系统后引起的急性神经元抑制反应

O'Rourke, Jacqueline G; Bachmann, Gemma; Mazur, Curt; Zhou, Keming; Platoshyn, Oleksandr; Bravo Hernandez, Mariana; Klein, Stephanie K; Nguyen, Jonathon; Burel, Sebastien; Hoffmaster, Christine; Zanardi, Thomas; Jafar-Nejad, Paymaan; Marsala, Martin; Henry, Scott P; Swayze, Eric E; Powers, Berit; Kordasiewicz, Holly B

Transient acute neuronal activation response caused by high concentrations of oligonucleotides in the cerebral spinal fluid

脑脊液中高浓度寡核苷酸引起的短暂急性神经元激活反应

Bravo-Hernandez, Mariana; Mazur, Curt; Chen, Hao; Fradkin, Linda; Searcy, Justin; Burel, Sebastien; Kelly, Mackenzie; Bruening, Dona; O'Rourke, Jacqueline G; Cai, Yuhang; Nguyen, Jonathon; Berman-Booty, Lisa; Cummins, Lendell; Gaus, Hans; Powers, Berit; Zhao, Hien T; Jafar-Nejad, Paymaan; Henry, Scott; Swayze, Eric E; Kordasiewicz, Holly B

Antisense oligonucleotide jacifusen for FUS-ALS: an investigator-initiated, multicentre, open-label case series

针对FUS-ALS的反义寡核苷酸jacifusen:一项研究者发起的多中心、开放标签病例系列研究

Shneider, Neil A; Harms, Matthew B; Korobeynikov, Vlad A; Rifai, Olivia M; Hoover, Benjamin N; Harrington, Elizabeth A; Aziz-Zaman, Sonya; Singleton, Jessica; Jamil, Arish; Madan, Vikram R; Lee, Ikjae; Andrews, Jinsy A; Smiley, Richard M; Alam, Mahabub M; Black, Lauren E; Shin, Minwook; Watts, Jonathan K; Walk, David; Newman, Daniel; Pascuzzi, Robert M; Weber, Markus; Neuwirth, Christoph; Da Cruz, Sandrine; Soriano, Armand; Lane, Roger; Henry, Scott; Mathews, Joel; Jafar-Nejad, Paymaan; Norris, Dan; Rigo, Frank; Brown, Robert H; Miller, Stephan; Crean, Rebecca; Bennett, C Frank

Therapeutic antisense oligonucleotide mitigates retinal dysfunction in a pig model of CLN3 Batten disease

治疗性反义寡核苷酸可减轻CLN3巴顿病猪模型中的视网膜功能障碍

Stratton, Matthew P; Centa, Jessica L; Swier, Vicki J; Pfeifer, Wanda L; Booth, Clarissa D; Albert, Karlee; Hunyara, John L; Rechtzigel, Mitchell J; Duelli, Fox J; Leppert, Hannah G; Rigo, Frank; Smit, Trisha; Jafar-Nejad, Paymaan; Weimer, Jill M; Drack, Arlene V; Hastings, Michelle L

Conjugation to a transferrin receptor 1-binding Bicycle peptide enhances ASO and siRNA potency in skeletal and cardiac muscles

与转铁蛋白受体1结合的双环肽偶联可增强ASO和siRNA在骨骼肌和心肌中的效力。

Østergaard, Michael E; Carrer, Michele; Anderson, Brooke A; Afetian, Megan; Bakooshli, Mohsen A; Santos, Jinro A; Klein, Stephanie K; Capitanio, Juliana; Freestone, Graeme C; Tanowitz, Michael; Galindo-Murillo, Rodrigo; Gaus, Hans J; Dwyer, Chrissa A; Jackson, Michaela; Jafar-Nejad, Paymaan; Rigo, Frank; Seth, Punit P; Gaynor, Katherine U; Stanway, Steven J; Urbonas, Liudvikas; St Denis, Megan A; Pellegrino, Simone; Bezerra, Gustavo A; Rigby, Michael; Gowans, Ellen; Van Rietschoten, Katerine; Beswick, Paul; Chen, Liuhong; Skynner, Michael J; Swayze, Eric E

Skeletal muscle effects of antisense oligonucleotides targeting glycogen synthase 1 in a mouse model of Pompe disease.

针对庞贝氏病小鼠模型中糖原合成酶 1 的反义寡核苷酸对骨骼肌的影响

Weiss Lan, Carrer Michele, Shmara Alyaa, Martin Angela, Yin Hong, Pal Pallabi, Cheng Cheng, Ta Lac, Boock Victoria, Fazeli Yasamin, Chang Mindy, Paguio Marvin, Lee Jonathan, Yu Howard, Weiss John, Grossman Tamar R, Raben Nina, Jafar-Nejad Paymaan, Kimonis Virginia

Targeting mechanistic target of rapamycin complex 2 attenuates immunopathology in systemic lupus erythematosus.

靶向雷帕霉素靶蛋白复合物 2 可减轻系统性红斑狼疮的免疫病理

Ai Minji, Zhou Xian, Carrer Michele, Jafar-Nejad Paymaan, Li Yanfeng, Gades Naomi, Alexander Mariam, Bautista-Vargas Mario, Duarte-García Alí, Zeng Hu

The differential impact of HNRNPA1 isoforms on gene expression and their relevance to dsRNA-mediated innate immune response.

HNRNPA1 同工型对基因表达的不同影响及其与 dsRNA 介导的先天免疫反应的相关性

Deshaies Jade-Emmanuelle, Triassi Valérie, Lacombe Andréanne, Gagné Myriam, Ling Karen, Ghosh Asmita, Labrecque Marjorie, Rigo Frank, Jafar-Nejad Paymaan, Tétreault Martine, Vande Velde Christine

A NOTCH3 pathogenic variant influences osteogenesis and can be targeted by antisense oligonucleotides in induced pluripotent stem cells

NOTCH3致病变异会影响成骨作用,并且可以通过诱导多能干细胞中的反义寡核苷酸进行靶向治疗。

Canalis, Ernesto; Yu, Jungeun; Schilling, Lauren; Jafar-Nejad, Paymaan; Carrer, Michele