A blood-brain penetrant RNA-targeted small molecule triggers elimination of r(G4C2)exp in c9ALS/FTD via the nuclear RNA exosome.
Bush JA, Meyer SM, Fuerst R, Tong Y, Li Y, Benhamou RI, Aikawa H, Zanon PRA, Gibaut QMR, Angelbello AJ, Gendron TF, Zhang YJ, Petrucelli L, Heick Jensen T, Childs-Disney JL, Disney MD.
Bush JA, et al. Among authors: benhamou ri.
Proc Natl Acad Sci U S A. 2022 Nov 29;119(48):e2210532119. doi: 10.1073/pnas.2210532119. Epub 2022 Nov 21.
Proc Natl Acad Sci U S A. 2022.
PMID: 36409902
Free PMC article.