A new patient-derived iPSC model for dystroglycanopathies validates a compound that increases glycosylation of α-dystroglycan.

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第一作者： Jihee,Kim

第一单位： Centre for Genomics and Child Health, Blizard Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, London, UK.;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.

作者： Jihee,Kim ^[1] ; Beatrice,Lana ^[1] ; Silvia,Torelli ^[2] ; David,Ryan ^[3] ; Francesco,Catapano ^[2] ; Pierpaolo,Ala ^[2] ; Christin,Luft ^[4] ; Elizabeth,Stevens ^[2] ; Evangelos,Konstantinidis ^[1] ; Sandra,Louzada ^[3] ; Beiyuan,Fu ^[3] ; Amaia,Paredes-Redondo ^[1] ; Aw Edith,Chan ^[5] ; Fengtang,Yang ^[3] ; Derek L,Stemple ^[3] ; Pentao,Liu ^[3] ; Robin,Ketteler ^[4] ; David L,Selwood ^[5] ; Francesco,Muntoni ^[6] ; Yung-Yao,Lin ^[1]

作者单位： Centre for Genomics and Child Health, Blizard Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, London, UK.;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. ^[1] UCL Great Ormond Street Institute of Child Health, London, UK. ^[2] Wellcome Sanger Institute, Hinxton, Cambridge, UK. ^[3] MRC Laboratory for Molecular Cell Biology, University College London, London, UK. ^[4] The Wolfson Institute for Biomedical Research, University College London, London, UK. ^[5] UCL Great Ormond Street Institute of Child Health, London, UK.;NIHR Biomedical Research Centre at Great Ormond Street Hospital, London, UK. ^[6]

关键词 CRISPR fukutin-related protein high-throughput screening human-induced pluripotent stem cells α-dystroglycan