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High-throughput screen of 100 000 small molecules in C9ORF72 ALS neurons identifies spliceosome modulators that mobilize G4C2 repeat RNA into nuclear export and repeat associated non-canonical translation.
Luteijn MJ, Bhaskar V, Trojer D, Schürz M, Mahboubi H, Handl C, Pizzato N, Pfeifer M, Dafinca R, Voshol H, Giorgetti E, Manneville C, Garnier IPM, Müller M, Zeng F, Buntin K, Markwalder R, Schröder H, Weiler J, Khar D, Schuhmann T, Groot-Kormelink PJ, Keller CG, Farmer P, MacKay A, Beibel M, Roma G, D'Ario G, Merkl C, Schebesta M, Hild M, Elwood F, Vahsen BF, Ripin N, Clery A, Allain F, Labow M, Gabriel D, Chao JA, Talbot K, Nash M, Hunziker J, Meisner-Kober NC. Luteijn MJ, et al. Among authors: d ario g, gabriel d. Nucleic Acids Res. 2025 Apr 10;53(7):gkaf253. doi: 10.1093/nar/gkaf253. Nucleic Acids Res. 2025. PMID: 40207633 Free PMC article.
603 results