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.
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| Title: | 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. |
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| Authors: | Luteijn MJ; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland., Bhaskar V; Friedrich Miescher Institute for Biomedical Research, Department Genomic Regulation, Basel, 4056, Switzerland., Trojer D; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland., Schürz M; Paris-Lodron University of Salzburg, Department of Biosciences and Medical Biology,, Salzburg, 5020,Austria.; Ludwig Boltzmann Institute for Nanovesicular Precision Medicine at the Paris Lodron University Salzburg, 5020, Austria., Mahboubi H; Friedrich Miescher Institute for Biomedical Research, Department Genomic Regulation, Basel, 4056, Switzerland., Handl C; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland., Pizzato N; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland., Pfeifer M; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland., Dafinca R; University of Oxford, John Radcliffe Hospital, Nuffield Department of Clinical Neurosciences, Oxford, OX3 9DU, United Kingdom., Voshol H; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland., Giorgetti E; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland., Manneville C; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland., Garnier IPM; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland., Müller M; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland., Zeng F; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland., Buntin K; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland., Markwalder R; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland., Schröder H; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland., Weiler J; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland., Khar D; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland., Schuhmann T; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland., Groot-Kormelink PJ; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland., Keller CG; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland., Farmer P; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland., MacKay A; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland., Beibel M; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland., Roma G; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland., D'Ario G; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland., Merkl C; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland., Schebesta M; Novartis Institutes for Biomedical Research, Department Discovery Sciences, Cambridge, MA02139, United States., Hild M; Novartis Institutes for Biomedical Research, Department Discovery Sciences, Cambridge, MA02139, United States., Elwood F; Novartis Institutes for Biomedical Research, Department Discovery Sciences, Cambridge, MA02139, United States., Vahsen BF; ETH Zürich, Department of Biology, Institute f. Molekularbiol.u.Biophysik, Zürich, 8093, Switzerland., Ripin N; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.; ETH Zürich, Department of Biology, Institute f. Molekularbiol.u.Biophysik, Zürich, 8093, Switzerland., Clery A; ETH Zürich, Department of Biology, Institute f. Molekularbiol.u.Biophysik, Zürich, 8093, Switzerland., Allain F; ETH Zürich, Department of Biology, Institute f. Molekularbiol.u.Biophysik, Zürich, 8093, Switzerland., Labow M; Novartis Institutes for Biomedical Research, Department Discovery Sciences, Cambridge, MA02139, United States., Gabriel D; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland., Chao JA; Friedrich Miescher Institute for Biomedical Research, Department Genomic Regulation, Basel, 4056, Switzerland., Talbot K; University of Oxford, John Radcliffe Hospital, Nuffield Department of Clinical Neurosciences, Oxford, OX3 9DU, United Kingdom., Nash M; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland., Hunziker J; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland., Meisner-Kober NC; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.; Paris-Lodron University of Salzburg, Department of Biosciences and Medical Biology,, Salzburg, 5020,Austria.; Ludwig Boltzmann Institute for Nanovesicular Precision Medicine at the Paris Lodron University Salzburg, 5020, Austria. |
| Source: | Nucleic acids research [Nucleic Acids Res] 2025 Apr 10; Vol. 53 (7). |
| Publication Type: | Journal Article |
| Journal Info: | Publisher: Oxford University Press Country of Publication: England NLM ID: 0411011 Publication Model: Print Cited Medium: Internet ISSN: 1362-4962 (Electronic) Linking ISSN: 03051048 NLM ISO Abbreviation: Nucleic Acids Res Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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| ISSN: | 1362-4962 |
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| DOI: | 10.1093/nar/gkaf253 |
