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 journalArticlepeer-review

Original languageEnglish
JournalProceedings of the National Academy of Sciences of the United States of America
Issue number119
Publication statusPublished - 21 Nov 2022

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