Original language | English |
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Journal | Proceedings of the National Academy of Sciences of the United States of America |
Volume | 2022 |
Issue number | 119 |
DOIs | |
Publication status | Published - 21 Nov 2022 |
A blood–brain penetrant RNA-targeted small molecule triggers elimination of r(G4C2)exp in c9ALS/FTD via the nuclear RNA exosome
Jessica A. Bush, Samantha M. Meyer, Rita Fürst, Yuquan Tong, Yue Li, Raphael I. Benhamou, Haruo Aikawa, Patrick R. A. Zanon, Quentin M. R. Gibaut, Alicia J. Angelbello, Tania F. Gendron, Yongjie Zhang, Leonard Petrucelli, Torben Heick Jensen, Jessica L. Childs-Disney, Matthew D. Disney*
*Corresponding author for this work
Research output: Contribution to journal › Article › peer-review