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 Res · 2025 · PMC11983130 · PMID 40207633

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highly significantno p-value reported

Furthermore, log(Heavy/Light) ratios of SRSF1 and SF3B1 IPs for proteins that are present in both samples largely overlapped, as visualized by the highly significant correlation depicted in Supplementary Fig.

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