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
期刊:Science
影响因子:45.8
doi:10.1126/science.adv2600
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