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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.
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: gendron tf. 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.
FUS regulates RAN translation through modulating the G-quadruplex structure of GGGGCC repeat RNA in C9orf72-linked ALS/FTD.
Fujino Y, Ueyama M, Ishiguro T, Ozawa D, Ito H, Sugiki T, Murata A, Ishiguro A, Gendron T, Mori K, Tokuda E, Taminato T, Konno T, Koyama A, Kawabe Y, Takeuchi T, Furukawa Y, Fujiwara T, Ikeda M, Mizuno T, Mochizuki H, Mizusawa H, Wada K, Ishikawa K, Onodera O, Nakatani K, Petrucelli L, Taguchi H, Nagai Y. Fujino Y, et al. Among authors: gendron t. Elife. 2023 Jul 18;12:RP84338. doi: 10.7554/eLife.84338. Elife. 2023. PMID: 37461319 Free PMC article.
Moderate intrinsic phenotypic alterations in C9orf72 ALS/FTD iPSC-microglia despite the presence of C9orf72 pathological features.
Lorenzini I, Alsop E, Levy J, Gittings LM, Lall D, Rabichow BE, Moore S, Pevey R, Bustos LM, Burciu C, Bhatia D, Singer M, Saul J, McQuade A, Tzioras M, Mota TA, Logemann A, Rose J, Almeida S, Gao FB, Marks M, Donnelly CJ, Hutchins E, Hung ST, Ichida J, Bowser R, Spires-Jones T, Blurton-Jones M, Gendron TF, Baloh RH, Van Keuren-Jensen K, Sattler R. Lorenzini I, et al. Among authors: gendron tf. Front Cell Neurosci. 2023 Jun 6;17:1179796. doi: 10.3389/fncel.2023.1179796. eCollection 2023. Front Cell Neurosci. 2023. PMID: 37346371 Free PMC article.
186 results