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

Blocking RAN translation without altering repeat RNAs rescues C9ORF72-related ALS and FTD phenotypes

阻断 RAN 翻译而不改变重复 RNA 可挽救 C9ORF72 相关 ALS 和 FTD 表型

Jiang, Xin; Schaeffer, Laure; Patni, Divya; Russo, Tommaso; Lee, Chao-Zong; Aguilar, Corey; Marques, Christine; Jansen-West, Karen; Hruska-Plochan, Marian; Ray-Soni, Ananya; Lim, Su Min; Held, Aaron; Yue, Mei; Castellanos Otero, Paula; Aryal, Sandeep; Beaussant, Hortense D A M; Basu, Himanish; Takakuwa, Hiro; Daughrity, Lillian M; Ramesh, Nandini; Da Costa, Paulo; A A Quadros, Ana Rita; Nolan, Matthew; Reyes, Charles Jourdan F; Wheeler, Hayden; Moran, Laura C; Griesman, Grant; Wymann, Benjamin; Trombetta, Bianca A; Lopez-De-Silanes, Emma Sofia; Canori, Michael; Krishnan, Gopinath; Vieira Souza Da Silva, Yasmim; Eriani, Gilbert; Albers, Mark W; Arnold, Steven E; Song, Yuyu; Jain, Ankur; Chiu, Isaac M; Zhang, Yong-Jie; Gao, Fen-Biao; Wainger, Brian J; Polymenidou, Magdalini; Petrucelli, Leonard; Martin, Franck; Lagier-Tourenne, Clotilde

Large-scale drug screening in iPSC-derived motor neurons from sporadic ALS patients identifies a potential combinatorial therapy

对散发性ALS患者诱导多能干细胞衍生的运动神经元进行大规模药物筛选,发现了一种潜在的联合疗法

Bye, Christopher R; Qian, Elizabeth; Lim, Katherine; Daniszewski, Maciej; Garton, Fleur C; Trần-Lê, Bảo C; Liang, Helena H; Lin, Tian; Lock, John G; Crombie, Duncan E; Morgan, Steven; Hu, Yi; Barton, Samantha K; Palmer, Lucy M; Djouma, Elvan; Kodikara, Saritha; Lê Cao, Kim-Anh; Dharmadasa, Thanuja; Henders, Anjali K; Ziser, Laura A; Kiernan, Matthew C; Talbot, Kevin; Needham, Merrilee; Fletcher, Susan; Talman, Paul; Mathers, Susan; Wray, Naomi R; Hewitt, Alex W; Pebay, Alice; Turner, Bradley J

Axonal Eif5a hypusination controls local translation and mitigates defects in FUS-ALS.

轴突 Eif5a 低磷化控制局部翻译,减轻 FUS-ALS 的缺陷。

Piol Diana, Khalil Bilal, Robberechts Tessa, Killian Theo, Georgopoulou Maria, Partel Gabriele, Wouters David, Hecker Nikolai, Tziortzouda Paraskevi, Verresen Yana, Corthout Nikky, Kint Sam, Vandereyken Katy, Van Damme Philip, Voet Thierry, Davie Kristofer, Poovathingal Suresh, Van Den Bosch Ludo, Aerts Stein, Sifrim Alejandro, Da Cruz Sandrine

Impaired nucleocytoplasmic transport in SOD1-mediated ALS.

SOD1介导的ALS中核质转运受损。

Argueti-Ostrovsky Shirel, Lim Su Min, Arogundade Olubankole A, Diaz-Garcia Sandra, Yunisova Gulshan, Meng Alex, Hermann Anita, Ong Kailee, Eremenko Ekaterina, Bravo-Hernandez Mariana, Driscoll Shawn P, Lee Chao-Zong, Jiang Xin, Stavsky Alexandra, Barel Shir, Shani Tom, Kahn Joy, Pfaff Samuel L, Monsonego Alon, Marsala Martin, Ravits John, Lagier-Tourenne Clotilde, Israelson Adrian

Aberrant Splicing Signatures Underpin Oligodendrocyte Damage in ALS and Neuron Loss in FTD

异常剪接特征是肌萎缩侧索硬化症(ALS)中少突胶质细胞损伤和额颞叶痴呆(FTD)中神经元丢失的根本原因。

Du, Chen; Li, Yinming; Wu, Rong; Shen, Yufei; Yang, Jiayi; Xiao, Xuan; Zhou, Yu

RIG-I Mediated Neuron-Specific IFN Type 1 Signaling in FUS-ALS Induces Neurodegeneration and Offers New Biomarker-Driven Individualized Treatment Options for (FUS-)ALS.

RIG-I 介导的神经元特异性 IFN 1 型信号传导在 FUS-ALS 中诱导神经退行性变,并为 (FUS-)ALS 提供新的生物标志物驱动的个体化治疗选择。

Naumann Marcel, Wierschin Theresa M, Kretschmer Stefanie, Dash Banaja P, Held Aaron, Salzinger Andrea, Peikert Kevin, Karlek Anže, Glaß Hannes, Großmann Dajana, Günther René, Petri Susanne, Rödiger Annekathrin, Brenner David, Pan-Montojo Francisco, Aronica Eleonora, Kipp Markus, Zimyanin Vitaly, Sterneckert Jared, Grehl Torsten, Seebacher Noah D, Böckers Tobias M, Catanese Alberto, Wainger Brian J, Oeckl Patrick, Lee-Kirsch Min Ae, Hermann Andreas

Microglial HVCN1 Deficiency Improves Movement and Survival of SOD1(G93A) ALS Mice by Enhancing Microglial Migration and Neuroprotection.

小胶质细胞 HVCN1 缺乏通过增强小胶质细胞迁移和神经保护作用改善 SOD1(G93A) ALS 小鼠的运动和存活率。

Wang Fan, Zhang Ke-Yu, Zhu Lang-Jian, Li Wei-Jue, Wu Yang, Gao Xiang, Ma Xiao-Ru, Yin Xiu-Hua, Wu Jian-Bin, Ye Xiao-Kang, Dong Zhao-Jun, Wang Di-Xian, Zhou Zhe, Wang Shao-Dong, Han Lei, Jiang Zhi-Nong, Zhao Jing-Wei

Engineered GM1 Intersects Between Mitochondrial and Synaptic Pathways to Ameliorate ALS Pathology.

工程化GM1连接线粒体和突触通路,以改善ALS病理。

Pilotto Federica, Toth Tristan Dellazizzo, Bond Silvano, Schmitz Alexander, Diab Rim, Tenlep Sara Y Ngo, Mooney Brian, Erni Silvia, Schobesberger Martina, Scheidegger Olivier, Peitsch Camille, Saxena Smita

Transcriptomic Analysis Reveals the Beneficial Effects of Spermidine in an ALS Mouse Model

转录组分析揭示亚精胺在ALS小鼠模型中的有益作用

Tang, Maoxue; Teng, Sasa; Peng, Yueqing; Kim, Ashley Y; Her, Yoon-Ra; Canoll, Peter; Bruce, Jeffrey N; Faust, Phyllis L; Adhikari, Kailash; De Vivo, Darryl C; Monani, Umrao R; Akman, Nur; Kömüroğlu, Ahmet Ufuk; Çibuk, Salih; Altındağ, Fikret; Yılmaz, Osman; Ateşşahin, Ahmet; Fiorucci, Cristian; Rossi, Marianna Nicoletta; Santo, Rachele Di; Salvatori, Illari; Scaricamazza, Silvia; Giuliani, Stefano; Carletta, Olga; Filomena, Ermes; Laurenti, Davide; Mattioli, Roberto; Mosca, Luciana; Valle, Cristiana; Ferri, Alberto; D’Erchia, Anna Maria; Cervelli, Manuela

Splicing the narrative: alternative TARDBP splicing and its relation to neurodegeneration in ALS and FTD

叙事拼接:TARDBP 选择性剪接及其与 ALS 和 FTD 神经退行性变的关系

Miller, Morgan R; Dykstra, Megan; Barmada, Sami