Ribonuclease recruitment using a small molecule reduced c9ALS/FTD r(G4C2) repeat expansion in vitro and in vivo ALS models
使用小分子进行核糖核酸酶募集可减少体外和体内 ALS 模型中的 c9ALS/FTD r(G4C2) 重复扩增
期刊:Science Translational Medicine
影响因子:15.8
doi:10.1126/scitranslmed.abd5991
Jessica A Bush, Haruo Aikawa, Rita Fuerst, Yue Li, Andrei Ursu, Samantha M Meyer, Raphael I Benhamou, Jonathan L Chen, Tanya Khan, Sarah Wagner-Griffin, Montina J Van Meter, Yuquan Tong, Hailey Olafson, Kendra K McKee, Jessica L Childs-Disney, Tania F Gendron, Yongjie Zhang, Alyssa N Coyne, Eric T W