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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 Fuerst, Yuquan Tong, Yue Li, Raphael I. Benhamou, Haruo Aikawa, Patrick R.A. Zanon, Quentin M.R. Gibaut, Alicia J. Angelbello, Tania F. Gendron, Yong Jie Zhang, Leonard Petrucelli, Torben Heick Jensen, Jessica L. Childs-Disney, Matthew D. Disney
Research output: Contribution to journal › Article › peer-review