1区 · 医学
Article
作者: Zhang, Yongjie ; Khan, Tanya ; Fuerst, Rita ; Tong, Yuquan ; Meyer, Samantha M. ; Disney, Matthew D. ; Rothstein, Jeffrey D. ; Chen, Jonathan L. ; Wagner-Griffin, Sarah ; Bush, Jessica A. ; Childs-Disney, Jessica L. ; Yildirim, Ilyas ; Aikawa, Haruo ; Ursu, Andrei ; Wang, Kye Won ; Coyne, Alyssa N. ; Li, Yue ; Gendron, Tania F. ; Petrucelli, Leonard ; Wang, Eric T. ; Benhamou, Raphael I. ; McKee, Kendra K. ; Olafson, Hailey ; Van Meter, Montina J.
Small-molecule recruitment of an endogenous nuclease rescues pathologies associated with c9ALS/FTD in vivo by targeting an RNA repeat expansion.