Generation of Induced Pluripotent Stem Cells From Patients With Duchenne Muscular Dystrophy and Their Induction to Cardiomyocytes

  • Hashimoto Akihito
    Department of Cardiovascular Regenerative Medicine, Osaka University Graduate School of Medicine Department of Cardiovascular Medicine, Osaka University Graduate School of Medicine
  • Naito Atsuhiko T.
    Department of Cardiovascular Regenerative Medicine, Osaka University Graduate School of Medicine Department of Cardiovascular Medicine, Graduate School of Medicine, The University of Tokyo
  • Lee Jong-Kook
    Department of Cardiovascular Regenerative Medicine, Osaka University Graduate School of Medicine Department of Cardiovascular Medicine, Osaka University Graduate School of Medicine
  • Kitazume-Taneike Rika
    Department of Cardiovascular Medicine, Osaka University Graduate School of Medicine
  • Ito Masamichi
    Department of Cardiovascular Medicine, Graduate School of Medicine, The University of Tokyo
  • Yamaguchi Toshihiro
    Department of Cardiovascular Medicine, Graduate School of Medicine, The University of Tokyo
  • Nakata Ryo
    Department of Cardiovascular Medicine, Graduate School of Medicine, The University of Tokyo
  • Sumida Tomokazu
    Department of Cardiovascular Medicine, Graduate School of Medicine, The University of Tokyo
  • Okada Katsuki
    Department of Cardiovascular Regenerative Medicine, Osaka University Graduate School of Medicine Department of Cardiovascular Medicine, Osaka University Graduate School of Medicine
  • Nakagawa Akito
    Department of Cardiovascular Regenerative Medicine, Osaka University Graduate School of Medicine Department of Cardiovascular Medicine, Osaka University Graduate School of Medicine
  • Higo Tomoaki
    Department of Cardiovascular Regenerative Medicine, Osaka University Graduate School of Medicine Department of Cardiovascular Medicine, Osaka University Graduate School of Medicine
  • Kuramoto Yuki
    Department of Cardiovascular Regenerative Medicine, Osaka University Graduate School of Medicine Department of Cardiovascular Medicine, Osaka University Graduate School of Medicine
  • Sakai Taku
    Department of Cardiovascular Regenerative Medicine, Osaka University Graduate School of Medicine Department of Cardiovascular Medicine, Osaka University Graduate School of Medicine
  • Tominaga Koji
    Department of Pediatrics
  • Okinaga Takeshi
    Department of Pediatrics
  • Kogaki Shigetoyo
    Department of Pediatrics
  • Ozono Keiichi
    Department of Pediatrics
  • Miyagawa Shigeru
    Department of Cardiovascular Surgery, Osaka University Graduate School of Medicine
  • Sawa Yoshiki
    Department of Cardiovascular Surgery, Osaka University Graduate School of Medicine
  • Sakata Yasushi
    Department of Cardiovascular Medicine, Osaka University Graduate School of Medicine
  • Morita Hiroyuki
    Department of Cardiovascular Medicine, Graduate School of Medicine, The University of Tokyo
  • Umezawa Akihiro
    Department of Reproductive Biology, Center for Regenerative Medicine, National Research Institute for Child Health and Development
  • Komuro Issei
    Department of Cardiovascular Medicine, Graduate School of Medicine, The University of Tokyo Department of Institute for Academic Initiatives, Osaka University

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Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene which encodes dystrophin protein. Dystrophin defect affects cardiac muscle as well as skeletal muscle. Cardiac dysfunction is observed in all patients with DMD over 18 years of age, but there is no curative treatment for DMD cardiomyopathy. To establish novel experimental platforms which reproduce the cardiac phenotype of DMD patients, here we established iPS cell lines from T lymphocytes donated from two DMD patients, with a protocol using Sendai virus vectors. We successfully conducted the differentiation of the DMD patient-specific iPS cells into beating cardiomyocytes. DMD patient-specific iPS cells and iPS cell-derived cardiomyocytes would be a useful in vitro experimental system with which to investigate DMD cardiomyopathy.

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