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An alternative splicing modulator decreases mutant HTT and improves the molecular fingerprint in Huntington's disease patient neurons.
Krach, Florian; Stemick, Judith; Boerstler, Tom; Weiss, Alexander; Lingos, Ioannis; Reischl, Stephanie; Meixner, Holger; Ploetz, Sonja; Farrell, Michaela; Hehr, Ute; Kohl, Zacharias; Winner, Beate; Winkler, Juergen.
Afiliação
  • Krach F; Department of Stem Cell Biology, University Hospital Erlangen, Friedrich-Alexander University of Erlangen-Nürnberg (FAU), Erlangen, Germany.
  • Stemick J; Department of Molecular Neurology, University Hospital Erlangen, Friedrich-Alexander University of Erlangen-Nürnberg (FAU), Erlangen, Germany.
  • Boerstler T; Department of Stem Cell Biology, University Hospital Erlangen, Friedrich-Alexander University of Erlangen-Nürnberg (FAU), Erlangen, Germany.
  • Weiss A; Evotec SE, Hamburg, Germany.
  • Lingos I; Evotec SE, Hamburg, Germany.
  • Reischl S; Department of Stem Cell Biology, University Hospital Erlangen, Friedrich-Alexander University of Erlangen-Nürnberg (FAU), Erlangen, Germany.
  • Meixner H; Department of Molecular Neurology, University Hospital Erlangen, Friedrich-Alexander University of Erlangen-Nürnberg (FAU), Erlangen, Germany.
  • Ploetz S; Department of Molecular Neurology, University Hospital Erlangen, Friedrich-Alexander University of Erlangen-Nürnberg (FAU), Erlangen, Germany.
  • Farrell M; Department of Stem Cell Biology, University Hospital Erlangen, Friedrich-Alexander University of Erlangen-Nürnberg (FAU), Erlangen, Germany.
  • Hehr U; Zentrum für Humangenetik Regensburg, Regensburg, Germany.
  • Kohl Z; Department of Neurology, University of Regensburg, Regensburg, Germany.
  • Winner B; Department of Stem Cell Biology, University Hospital Erlangen, Friedrich-Alexander University of Erlangen-Nürnberg (FAU), Erlangen, Germany. beate.winner@fau.de.
  • Winkler J; Center for Rare Diseases Erlangen (ZSEER), University Hospital Erlangen, Friedrich-Alexander University of Erlangen-Nürnberg (FAU), Erlangen, Germany. beate.winner@fau.de.
Nat Commun ; 13(1): 6797, 2022 11 10.
Article em En | MEDLINE | ID: mdl-36357392
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Doença de Huntington Tipo de estudo: Prognostic_studies Limite: Humans Idioma: En Revista: Nat Commun Assunto da revista: BIOLOGIA / CIENCIA Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Alemanha
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Doença de Huntington Tipo de estudo: Prognostic_studies Limite: Humans Idioma: En Revista: Nat Commun Assunto da revista: BIOLOGIA / CIENCIA Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Alemanha