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 (Nucleic Acids Research, 2025)

(untitled) - Luteijn, MJ; Bhaskar, V; Trojer, D; Schürz, M; Mahboubi, H; Handl, C; Pizzato, N; Pfeifer, M; Dafinca, R; Voshol, H; Giorgetti, E; Manneville, C; et al.