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

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 ca...

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Veröffentlicht in:International Heart Journal 2016, Vol.57(1), pp.112-117
Hauptverfasser: Hashimoto, Akihito, Naito, Atsuhiko T., Lee, Jong-Kook, Kitazume-Taneike, Rika, Ito, Masamichi, Yamaguchi, Toshihiro, Nakata, Ryo, Sumida, Tomokazu, Okada, Katsuki, Nakagawa, Akito, Higo, Tomoaki, Kuramoto, Yuki, Sakai, Taku, Tominaga, Koji, Okinaga, Takeshi, Kogaki, Shigetoyo, Ozono, Keiichi, Miyagawa, Shigeru, Sawa, Yoshiki, Sakata, Yasushi, Morita, Hiroyuki, Umezawa, Akihiro, Komuro, Issei
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container_issue 1
container_start_page 112
container_title International Heart Journal
container_volume 57
creator Hashimoto, Akihito
Naito, Atsuhiko T.
Lee, Jong-Kook
Kitazume-Taneike, Rika
Ito, Masamichi
Yamaguchi, Toshihiro
Nakata, Ryo
Sumida, Tomokazu
Okada, Katsuki
Nakagawa, Akito
Higo, Tomoaki
Kuramoto, Yuki
Sakai, Taku
Tominaga, Koji
Okinaga, Takeshi
Kogaki, Shigetoyo
Ozono, Keiichi
Miyagawa, Shigeru
Sawa, Yoshiki
Sakata, Yasushi
Morita, Hiroyuki
Umezawa, Akihiro
Komuro, Issei
description 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.
doi_str_mv 10.1536/ihj.15-376
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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. 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subjects Adolescent
Adult
Cardiomyopathy
Cell Differentiation
Cells, Cultured
Human
Humans
Induced Pluripotent Stem Cells - cytology
Induced Pluripotent Stem Cells - physiology
Muscular Dystrophy, Duchenne - genetics
Muscular Dystrophy, Duchenne - metabolism
Muscular Dystrophy, Duchenne - pathology
Myocytes, Cardiac - cytology
Myocytes, Cardiac - metabolism
Real-Time Polymerase Chain Reaction
RNA - genetics
title Generation of Induced Pluripotent Stem Cells From Patients With Duchenne Muscular Dystrophy and Their Induction to Cardiomyocytes
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