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Targeting RNA structure in SMN2 reverses spinal muscular atrophy molecular phenotypes.
Garcia-Lopez, Amparo; Tessaro, Francesca; Jonker, Hendrik R A; Wacker, Anna; Richter, Christian; Comte, Arnaud; Berntenis, Nikolaos; Schmucki, Roland; Hatje, Klas; Petermann, Olivier; Chiriano, Gianpaolo; Perozzo, Remo; Sciarra, Daniel; Konieczny, Piotr; Faustino, Ignacio; Fournet, Guy; Orozco, Modesto; Artero, Ruben; Metzger, Friedrich; Ebeling, Martin; Goekjian, Peter; Joseph, Benoît; Schwalbe, Harald; Scapozza, Leonardo.
Afiliação
  • Garcia-Lopez A; Pharmaceutical Biochemistry Group, School of Pharmaceutical Sciences, University of Lausanne and University of Geneva, Rue Michel-Servet 1, 1211, Geneva, Switzerland. amparo.garcialopez@unige.ch.
  • Tessaro F; Pharmaceutical Biochemistry Group, School of Pharmaceutical Sciences, University of Lausanne and University of Geneva, Rue Michel-Servet 1, 1211, Geneva, Switzerland.
  • Jonker HRA; Institut für Organische Chemie und Chemische Biologie, Center for Biomolecular Magnetic Resonance (BMRZ), Johann Wolfgang Goethe-University Frankfurt, Max-von-Laue-Strasse 7, 60438, Frankfurt, Germany.
  • Wacker A; Institut für Organische Chemie und Chemische Biologie, Center for Biomolecular Magnetic Resonance (BMRZ), Johann Wolfgang Goethe-University Frankfurt, Max-von-Laue-Strasse 7, 60438, Frankfurt, Germany.
  • Richter C; Institut für Organische Chemie und Chemische Biologie, Center for Biomolecular Magnetic Resonance (BMRZ), Johann Wolfgang Goethe-University Frankfurt, Max-von-Laue-Strasse 7, 60438, Frankfurt, Germany.
  • Comte A; Institut de Chimie et Biochimie Moléculaires et Supramoléculaires (ICBMS), UMR CNRS 5246, University Claude Bernard Lyon 1, 43 Bd du 11 Novembre 1918, F-69622, Villeurbanne cedex, France.
  • Berntenis N; Pharmaceutical Research and Early Development, F. Hoffmann-La Roche, Roche Innovation Center Basel, Grenzacherstrasse 124, 4070, Basel, Switzerland.
  • Schmucki R; Pharmaceutical Research and Early Development, F. Hoffmann-La Roche, Roche Innovation Center Basel, Grenzacherstrasse 124, 4070, Basel, Switzerland.
  • Hatje K; Pharmaceutical Research and Early Development, F. Hoffmann-La Roche, Roche Innovation Center Basel, Grenzacherstrasse 124, 4070, Basel, Switzerland.
  • Petermann O; Pharmaceutical Biochemistry Group, School of Pharmaceutical Sciences, University of Lausanne and University of Geneva, Rue Michel-Servet 1, 1211, Geneva, Switzerland.
  • Chiriano G; Pharmaceutical Biochemistry Group, School of Pharmaceutical Sciences, University of Lausanne and University of Geneva, Rue Michel-Servet 1, 1211, Geneva, Switzerland.
  • Perozzo R; Pharmaceutical Biochemistry Group, School of Pharmaceutical Sciences, University of Lausanne and University of Geneva, Rue Michel-Servet 1, 1211, Geneva, Switzerland.
  • Sciarra D; Pharmaceutical Biochemistry Group, School of Pharmaceutical Sciences, University of Lausanne and University of Geneva, Rue Michel-Servet 1, 1211, Geneva, Switzerland.
  • Konieczny P; Translational Genomics Group, Incliva Health Research Institute, Menendez Pelayo 4, 46010, Valencia, Spain.
  • Faustino I; Department of Genetics and Interdisciplinary Research Structure for Biotechnology and Biomedicine (ERI BIOTECMED), University of Valencia, Dr Moliner 50, 46100, Burjassot, Spain.
  • Fournet G; Institute for Research in Biomedicine (IRB), Barcelona Institute of Science and Technology (BIST), Joint BSC-IRB Research Program in Computational Biology, Baldiri Reixac 10, 08028, Barcelona, Spain.
  • Orozco M; Institut de Chimie et Biochimie Moléculaires et Supramoléculaires (ICBMS), UMR CNRS 5246, University Claude Bernard Lyon 1, 43 Bd du 11 Novembre 1918, F-69622, Villeurbanne cedex, France.
  • Artero R; Institute for Research in Biomedicine (IRB), Barcelona Institute of Science and Technology (BIST), Joint BSC-IRB Research Program in Computational Biology, Baldiri Reixac 10, 08028, Barcelona, Spain.
  • Metzger F; Translational Genomics Group, Incliva Health Research Institute, Menendez Pelayo 4, 46010, Valencia, Spain.
  • Ebeling M; Department of Genetics and Interdisciplinary Research Structure for Biotechnology and Biomedicine (ERI BIOTECMED), University of Valencia, Dr Moliner 50, 46100, Burjassot, Spain.
  • Goekjian P; Pharmaceutical Research and Early Development, F. Hoffmann-La Roche, Roche Innovation Center Basel, Grenzacherstrasse 124, 4070, Basel, Switzerland.
  • Joseph B; Pharmaceutical Research and Early Development, F. Hoffmann-La Roche, Roche Innovation Center Basel, Grenzacherstrasse 124, 4070, Basel, Switzerland.
  • Schwalbe H; Institut de Chimie et Biochimie Moléculaires et Supramoléculaires (ICBMS), UMR CNRS 5246, University Claude Bernard Lyon 1, 43 Bd du 11 Novembre 1918, F-69622, Villeurbanne cedex, France.
  • Scapozza L; Institut de Chimie et Biochimie Moléculaires et Supramoléculaires (ICBMS), UMR CNRS 5246, University Claude Bernard Lyon 1, 43 Bd du 11 Novembre 1918, F-69622, Villeurbanne cedex, France.
Nat Commun ; 9(1): 2032, 2018 05 23.
Article em En | MEDLINE | ID: mdl-29795225
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: RNA Mensageiro / Atrofia Muscular Espinal / Imidazóis / Indóis Idioma: En Revista: Nat Commun Ano de publicação: 2018 Tipo de documento: Article País de afiliação: Suíça
Texto completo
Adicionar na Minha BVS
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: RNA Mensageiro / Atrofia Muscular Espinal / Imidazóis / Indóis Idioma: En Revista: Nat Commun Ano de publicação: 2018 Tipo de documento: Article País de afiliação: Suíça