View in EDS HTML Full Text PDF Full Text

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.

Saved in:
Bibliographic Details
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.
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
Full text is not displayed to guests.
FullText Links:
  – Type: pdflink
Text:
  Availability: 1
Header DbId: mdl
DbLabel: MEDLINE Ultimate
An: 40207633
AccessLevel: 2
PubType: Academic Journal
PubTypeId: academicJournal
PreciseRelevancyScore: 0
IllustrationInfo
Items – Name: Title
  Label: Title
  Group: Ti
  Data: 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.
– Name: Author
  Label: Authors
  Group: Au
  Data: <searchLink fieldCode="AU" term="%22Luteijn+MJ%22">Luteijn MJ</searchLink>; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Bhaskar+V%22">Bhaskar V</searchLink>; Friedrich Miescher Institute for Biomedical Research, Department Genomic Regulation, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Trojer+D%22">Trojer D</searchLink>; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Schürz+M%22">Schürz M</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Mahboubi+H%22">Mahboubi H</searchLink>; Friedrich Miescher Institute for Biomedical Research, Department Genomic Regulation, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Handl+C%22">Handl C</searchLink>; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Pizzato+N%22">Pizzato N</searchLink>; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Pfeifer+M%22">Pfeifer M</searchLink>; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Dafinca+R%22">Dafinca R</searchLink>; University of Oxford, John Radcliffe Hospital, Nuffield Department of Clinical Neurosciences, Oxford, OX3 9DU, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Voshol+H%22">Voshol H</searchLink>; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Giorgetti+E%22">Giorgetti E</searchLink>; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Manneville+C%22">Manneville C</searchLink>; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Garnier+IPM%22">Garnier IPM</searchLink>; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Müller+M%22">Müller M</searchLink>; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Zeng+F%22">Zeng F</searchLink>; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Buntin+K%22">Buntin K</searchLink>; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Markwalder+R%22">Markwalder R</searchLink>; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Schröder+H%22">Schröder H</searchLink>; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Weiler+J%22">Weiler J</searchLink>; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Khar+D%22">Khar D</searchLink>; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Schuhmann+T%22">Schuhmann T</searchLink>; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Groot-Kormelink+PJ%22">Groot-Kormelink PJ</searchLink>; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Keller+CG%22">Keller CG</searchLink>; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Farmer+P%22">Farmer P</searchLink>; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22MacKay+A%22">MacKay A</searchLink>; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Beibel+M%22">Beibel M</searchLink>; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Roma+G%22">Roma G</searchLink>; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22D'Ario+G%22">D'Ario G</searchLink>; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Merkl+C%22">Merkl C</searchLink>; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Schebesta+M%22">Schebesta M</searchLink>; Novartis Institutes for Biomedical Research, Department Discovery Sciences, Cambridge, MA02139, United States.<br /><searchLink fieldCode="AU" term="%22Hild+M%22">Hild M</searchLink>; Novartis Institutes for Biomedical Research, Department Discovery Sciences, Cambridge, MA02139, United States.<br /><searchLink fieldCode="AU" term="%22Elwood+F%22">Elwood F</searchLink>; Novartis Institutes for Biomedical Research, Department Discovery Sciences, Cambridge, MA02139, United States.<br /><searchLink fieldCode="AU" term="%22Vahsen+BF%22">Vahsen BF</searchLink>; ETH Zürich, Department of Biology, Institute f. Molekularbiol.u.Biophysik, Zürich, 8093, Switzerland.<br /><searchLink fieldCode="AU" term="%22Ripin+N%22">Ripin N</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Clery+A%22">Clery A</searchLink>; ETH Zürich, Department of Biology, Institute f. Molekularbiol.u.Biophysik, Zürich, 8093, Switzerland.<br /><searchLink fieldCode="AU" term="%22Allain+F%22">Allain F</searchLink>; ETH Zürich, Department of Biology, Institute f. Molekularbiol.u.Biophysik, Zürich, 8093, Switzerland.<br /><searchLink fieldCode="AU" term="%22Labow+M%22">Labow M</searchLink>; Novartis Institutes for Biomedical Research, Department Discovery Sciences, Cambridge, MA02139, United States.<br /><searchLink fieldCode="AU" term="%22Gabriel+D%22">Gabriel D</searchLink>; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Chao+JA%22">Chao JA</searchLink>; Friedrich Miescher Institute for Biomedical Research, Department Genomic Regulation, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Talbot+K%22">Talbot K</searchLink>; University of Oxford, John Radcliffe Hospital, Nuffield Department of Clinical Neurosciences, Oxford, OX3 9DU, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Nash+M%22">Nash M</searchLink>; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Hunziker+J%22">Hunziker J</searchLink>; Novartis Institutes for Biomedical Research, Department Global Discovery Chemistry, Basel, 4056, Switzerland.<br /><searchLink fieldCode="AU" term="%22Meisner-Kober+NC%22">Meisner-Kober NC</searchLink>; 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.
– Name: TitleSource
  Label: Source
  Group: Src
  Data: <searchLink fieldCode="JN" term="%220411011%22">Nucleic acids research</searchLink> [Nucleic Acids Res] 2025 Apr 10; Vol. 53 (7).
– Name: TypePub
  Label: Publication Type
  Group: TypPub
  Data: Journal Article
– Name: TitleSource
  Label: Journal Info
  Group: Src
  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Oxford+University+Press%22">Oxford University Press </searchLink><i>Country of Publication: </i>England <i>NLM ID: </i>0411011 <i>Publication Model: </i>Print <i>Cited Medium: </i>Internet <i>ISSN: </i>1362-4962 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2203051048%22">03051048 </searchLink><i>NLM ISO Abbreviation: </i>Nucleic Acids Res <i>Subsets: </i>MEDLINE
PLink https://search.ebscohost.com/login.aspx?direct=true&site=eds-live&db=mdl&AN=40207633
RecordInfo BibRecord:
  BibEntity:
    Identifiers:
      – Type: doi
        Value: 10.1093/nar/gkaf253
    Languages:
      – Code: eng
        Text: English
    Titles:
      – TitleFull: 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.
        Type: main
  BibRelationships:
    HasContributorRelationships:
      – PersonEntity:
          Name:
            NameFull: Luteijn MJ
      – PersonEntity:
          Name:
            NameFull: Bhaskar V
      – PersonEntity:
          Name:
            NameFull: Trojer D
      – PersonEntity:
          Name:
            NameFull: Schürz M
      – PersonEntity:
          Name:
            NameFull: Mahboubi H
      – PersonEntity:
          Name:
            NameFull: Handl C
      – PersonEntity:
          Name:
            NameFull: Pizzato N
      – PersonEntity:
          Name:
            NameFull: Pfeifer M
      – PersonEntity:
          Name:
            NameFull: Dafinca R
      – PersonEntity:
          Name:
            NameFull: Voshol H
      – PersonEntity:
          Name:
            NameFull: Giorgetti E
      – PersonEntity:
          Name:
            NameFull: Manneville C
      – PersonEntity:
          Name:
            NameFull: Garnier IPM
      – PersonEntity:
          Name:
            NameFull: Müller M
      – PersonEntity:
          Name:
            NameFull: Zeng F
      – PersonEntity:
          Name:
            NameFull: Buntin K
      – PersonEntity:
          Name:
            NameFull: Markwalder R
      – PersonEntity:
          Name:
            NameFull: Schröder H
      – PersonEntity:
          Name:
            NameFull: Weiler J
      – PersonEntity:
          Name:
            NameFull: Khar D
      – PersonEntity:
          Name:
            NameFull: Schuhmann T
      – PersonEntity:
          Name:
            NameFull: Groot-Kormelink PJ
      – PersonEntity:
          Name:
            NameFull: Keller CG
      – PersonEntity:
          Name:
            NameFull: Farmer P
      – PersonEntity:
          Name:
            NameFull: MacKay A
      – PersonEntity:
          Name:
            NameFull: Beibel M
      – PersonEntity:
          Name:
            NameFull: Roma G
      – PersonEntity:
          Name:
            NameFull: D'Ario G
      – PersonEntity:
          Name:
            NameFull: Merkl C
      – PersonEntity:
          Name:
            NameFull: Schebesta M
      – PersonEntity:
          Name:
            NameFull: Hild M
      – PersonEntity:
          Name:
            NameFull: Elwood F
      – PersonEntity:
          Name:
            NameFull: Vahsen BF
      – PersonEntity:
          Name:
            NameFull: Ripin N
      – PersonEntity:
          Name:
            NameFull: Clery A
      – PersonEntity:
          Name:
            NameFull: Allain F
      – PersonEntity:
          Name:
            NameFull: Labow M
      – PersonEntity:
          Name:
            NameFull: Gabriel D
      – PersonEntity:
          Name:
            NameFull: Chao JA
      – PersonEntity:
          Name:
            NameFull: Talbot K
      – PersonEntity:
          Name:
            NameFull: Nash M
      – PersonEntity:
          Name:
            NameFull: Hunziker J
      – PersonEntity:
          Name:
            NameFull: Meisner-Kober NC
    IsPartOfRelationships:
      – BibEntity:
          Dates:
            – D: 10
              M: 04
              Text: 2025 Apr 10
              Type: published
              Y: 2025
          Identifiers:
            – Type: issn-electronic
              Value: 1362-4962
          Numbering:
            – Type: volume
              Value: 53
            – Type: issue
              Value: 7
          Titles:
            – TitleFull: Nucleic acids research
              Type: main
ResultId 1