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

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

Antisense oligonucleotide therapy for patients with Friedreich's ataxia carrying the c.165+5G>C splicing mutation.

针对携带 c.165+5G>C 剪接突变的 Friedreich 共济失调患者的反义寡核苷酸疗法

Yameogo Pouiré, Aguilar Selina, Prakash Thazha P, Rigo Frank, Lynch David R, Napierala Jill S, Napierala Marek

Synthesis, Incorporation, and Translation Properties of Threose Nucleic Acid Modified Trinucleotide Cap Analogs

苏糖核酸修饰的三核苷酸帽类似物的合成、掺入和翻译性质

Pontarelli, Alexander; Vasquez, Guillermo; Fu, Wuxia; Solan, Qingxia; Gaus, Hans J; Galindo-Murillo, Rodrigo; Huang, Pin-Yao; Pandit, Bhoomika; Rigo, Frank; Østergaard, Michael E; Freestone, Graeme C

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

Stathmin-2 loss leads to neurofilament-dependent axonal collapse driving motor and sensory denervation

Stathmin-2 的缺失会导致神经丝依赖性轴突塌陷,进而引发运动和感觉神经支配丧失。

López-Erauskin, Jone; Bravo-Hernandez, Mariana; Presa, Maximiliano; Baughn, Michael W; Melamed, Ze'ev; Beccari, Melinda S; Agra de Almeida Quadros, Ana Rita; Arnold-Garcia, Olatz; Zuberi, Aamir; Ling, Karen; Platoshyn, Oleksandr; Niño-Jara, Elkin; Ndayambaje, I Sandra; McAlonis-Downes, Melissa; Cabrera, Larissa; Artates, Jonathan W; Ryan, Jennifer; Hermann, Anita; Ravits, John; Bennett, C Frank; Jafar-Nejad, Paymaan; Rigo, Frank; Marsala, Martin; Lutz, Cathleen M; Cleveland, Don W; Lagier-Tourenne, Clotilde

Correction: Antisense oligonucleotide therapy rescues disturbed brain rhythms and sleep in juvenile and adult mouse models of Angelman syndrome

更正:反义寡核苷酸疗法可挽救幼年和成年安格曼综合征小鼠模型中紊乱的脑节律和睡眠。

Lee, Dongwon; Chen, Wu; Kaku, Heet Naresh; Zhuo, Xinming; Chao, Eugene S; Soriano, Armand; Kuncheria, Allen; Flores, Stephanie; Kim, Joo Hyun; Rivera, Armando; Rigo, Frank; Jafar-Nejad, Paymaan; Beaudet, Arthur L; Caudill, Matthew S; Xue, Mingshan

Elevating microRNA levels by targeting biogenesis with steric-blocking antisense oligonucleotides

利用空间位阻反义寡核苷酸靶向生物合成来提高microRNA水平

Havens, Mallory A; Hinrich, Anthony J; Rigo, Frank; Hastings, Michelle L