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A new patient-derived iPSC model for dystroglycanopathies validates a compound that increases glycosylation of α-dystroglycan.
Kim, Jihee; Lana, Beatrice; Torelli, Silvia; Ryan, David; Catapano, Francesco; Ala, Pierpaolo; Luft, Christin; Stevens, Elizabeth; Konstantinidis, Evangelos; Louzada, Sandra; Fu, Beiyuan; Paredes-Redondo, Amaia; Chan, Aw Edith; Yang, Fengtang; Stemple, Derek L; Liu, Pentao; Ketteler, Robin; Selwood, David L; Muntoni, Francesco; Lin, Yung-Yao.
Afiliación
  • Kim J; Centre for Genomics and Child Health, Blizard Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, London, UK.
  • Lana B; Stem Cell Laboratory, National Bowel Research Centre, Blizard Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, London, UK.
  • Torelli S; Centre for Genomics and Child Health, Blizard Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, London, UK.
  • Ryan D; Stem Cell Laboratory, National Bowel Research Centre, Blizard Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, London, UK.
  • Catapano F; UCL Great Ormond Street Institute of Child Health, London, UK.
  • Ala P; Wellcome Sanger Institute, Hinxton, Cambridge, UK.
  • Luft C; UCL Great Ormond Street Institute of Child Health, London, UK.
  • Stevens E; UCL Great Ormond Street Institute of Child Health, London, UK.
  • Konstantinidis E; MRC Laboratory for Molecular Cell Biology, University College London, London, UK.
  • Louzada S; UCL Great Ormond Street Institute of Child Health, London, UK.
  • Fu B; Centre for Genomics and Child Health, Blizard Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, London, UK.
  • Paredes-Redondo A; Stem Cell Laboratory, National Bowel Research Centre, Blizard Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, London, UK.
  • Chan AE; Wellcome Sanger Institute, Hinxton, Cambridge, UK.
  • Yang F; Wellcome Sanger Institute, Hinxton, Cambridge, UK.
  • Stemple DL; Centre for Genomics and Child Health, Blizard Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, London, UK.
  • Liu P; Stem Cell Laboratory, National Bowel Research Centre, Blizard Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, London, UK.
  • Ketteler R; The Wolfson Institute for Biomedical Research, University College London, London, UK.
  • Selwood DL; Wellcome Sanger Institute, Hinxton, Cambridge, UK.
  • Muntoni F; Wellcome Sanger Institute, Hinxton, Cambridge, UK.
  • Lin YY; Wellcome Sanger Institute, Hinxton, Cambridge, UK.
EMBO Rep ; 20(11): e47967, 2019 11 05.
Article en En | MEDLINE | ID: mdl-31566294

Texto completo: 1 Bases de datos: MEDLINE Asunto principal: Distroglicanos / Evaluación Preclínica de Medicamentos / Células Madre Pluripotentes Inducidas Tipo de estudio: Etiology_studies Límite: Humans Idioma: En Revista: EMBO Rep Asunto de la revista: BIOLOGIA MOLECULAR Año: 2019 Tipo del documento: Article País de afiliación: Reino Unido

Texto completo: 1 Bases de datos: MEDLINE Asunto principal: Distroglicanos / Evaluación Preclínica de Medicamentos / Células Madre Pluripotentes Inducidas Tipo de estudio: Etiology_studies Límite: Humans Idioma: En Revista: EMBO Rep Asunto de la revista: BIOLOGIA MOLECULAR Año: 2019 Tipo del documento: Article País de afiliación: Reino Unido