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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*
    • *Corresponding author for this work

Research output: Contribution to journal › Article › peer-review

23 Scopus citations

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Biochemistry, Genetics and Molecular Biology