Citations 1
Ribonuclease recruitment using a small molecule reduced c9ALS/FTD r(G 4 C 2 ) repeat expansion in vitro and in vivo ALS models
Bush, Jessica A. , Aikawa, Haruo , Fuerst, Rita, Li, Yue , Ursu, Andrei , Meyer, Samantha M. , Benhamou, Raphael I. , Chen, Jonathan L. , Khan, Tanya , Wagner-Griffin, Sarah , Meter, Montina J. Van , Tong, Yuquan , Olafson, Hailey, McKee, Kendra , Childs‐Disney, Jessica L. , Zhang, Yong‐Jie , Wang, Eric T. , Wang, Kye Won , Petrucelli, Leonard , Van Meter, Montina J , McKee, Kendra K , Gendron, Tania F , Zhang, Yong-Jie , Coyne, Alyssa N , Wang, Eric T , Wang, Kye Won , Petrucelli, Leonard , Rothstein, Jeffrey D , Disney, Matthew D , Benhamou, Raphael I , Aikawa, Haruo , Gendron, Tania F. , Coyne, Alyssa N. , Yildirim, Ilyas , Rothstein, Jeffrey D. , Disney, Matthew D.
Science Translational Medicine | October 27, 2021 | Volume: 13 | Issue: 617 | Pages: No page available
Small-molecule recruitment of an endogenous nuclease rescues pathologies associated with c9ALS/FTD in vivo by targeting an RNA repeat expansion.