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Combinatorial ASO-mediated therapy with low dose SMN and the protective modifier Chp1 is not sufficient to ameliorate SMA pathology hallmarks.
Muinos-Bühl, A; Rombo, R; Janzen, E; Ling, K K; Hupperich, K; Rigo, F; Bennett, C F; Wirth, B.
Afiliação
  • Muinos-Bühl A; Institute of Human Genetics, University of Cologne, 50931 Cologne, Germany; Center for Molecular Medicine Cologne, University of Cologne, 50931 Cologne, Germany; Institute for Genetics, University of Cologne, 50674 Cologne, Germany. Electronic address: anixa.muinos-buehl@uk-koeln.de.
  • Rombo R; Institute of Human Genetics, University of Cologne, 50931 Cologne, Germany; Center for Molecular Medicine Cologne, University of Cologne, 50931 Cologne, Germany; Institute for Genetics, University of Cologne, 50674 Cologne, Germany. Electronic address: roman.rombo@uk-koeln.de.
  • Janzen E; Institute of Human Genetics, University of Cologne, 50931 Cologne, Germany; Center for Molecular Medicine Cologne, University of Cologne, 50931 Cologne, Germany; Institute for Genetics, University of Cologne, 50674 Cologne, Germany.
  • Ling KK; Ionis Pharmaceuticals, Carlsbad, CA 92008, USA. Electronic address: kling@ionisph.com.
  • Hupperich K; Institute of Human Genetics, University of Cologne, 50931 Cologne, Germany; Center for Molecular Medicine Cologne, University of Cologne, 50931 Cologne, Germany; Institute for Genetics, University of Cologne, 50674 Cologne, Germany.
  • Rigo F; Ionis Pharmaceuticals, Carlsbad, CA 92008, USA. Electronic address: frigo@ionisph.com.
  • Bennett CF; Ionis Pharmaceuticals, Carlsbad, CA 92008, USA. Electronic address: fbennett@ionisph.com.
  • Wirth B; Institute of Human Genetics, University of Cologne, 50931 Cologne, Germany; Center for Molecular Medicine Cologne, University of Cologne, 50931 Cologne, Germany; Institute for Genetics, University of Cologne, 50674 Cologne, Germany; Center for Rare Diseases, University Hospital of Cologne, 50931 Col
Neurobiol Dis ; 171: 105795, 2022 09.
Article em En | MEDLINE | ID: mdl-35724821
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Atrofia Muscular Espinal Tipo de estudo: Prognostic_studies Limite: Animals Idioma: En Revista: Neurobiol Dis Ano de publicação: 2022 Tipo de documento: Article
Texto completo
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Atrofia Muscular Espinal Tipo de estudo: Prognostic_studies Limite: Animals Idioma: En Revista: Neurobiol Dis Ano de publicação: 2022 Tipo de documento: Article