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3. Modifiers of C9orf72 dipeptide repeat toxicity connect nucleocytoplasmic transport defects to FTD/ALS. Jovičić A; Mertens J; Boeynaems S; Bogaert E; Chai N; Yamada SB; Paul JW; Sun S; Herdy JR; Bieri G; Kramer NJ; Gage FH; Van Den Bosch L; Robberecht W; Gitler AD Nat Neurosci; 2015 Sep; 18(9):1226-9. PubMed ID: 26308983 [TBL] [Abstract][Full Text] [Related]
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5. BET bromodomain inhibitors PFI-1 and JQ1 are identified in an epigenetic compound screen to enhance C9ORF72 gene expression and shown to ameliorate C9ORF72-associated pathological and behavioral abnormalities in a C9ALS/FTD model. Quezada E; Cappelli C; Diaz I; Jury N; Wightman N; Brown RH; Montecino M; van Zundert B Clin Epigenetics; 2021 Mar; 13(1):56. PubMed ID: 33726839 [TBL] [Abstract][Full Text] [Related]
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