A blood-brain penetrant RNA-targeted small molecule triggers elimination of r(G4C2)exp in c9ALS/FTD via the nuclear RNA exosome
一种血脑渗透性 RNA 靶向小分子通过核 RNA 外泌体触发 c9ALS/FTD 中 r(G4C2)exp 的消除
期刊:Proceedings of the National Academy of Sciences of the United States of America
影响因子:
doi:10.1073/pnas.2210532119
Jessica A Bush, Samantha M Meyer, Rita Fuerst, Yuquan Tong, Yue Li, Raphael I Benhamou, Haruo Aikawa, Patrick R A Zanon, Quentin M R Gibaut, Alicia J Angelbello, Tania F Gendron, Yong-Jie Zhang, Leonard Petrucelli, Torben Heick Jensen, Jessica L Childs-Disney, Matthew D Disney