Contractile Activity of Myotubes Derived from Human Induced Pluripotent Stem Cells: A Model of Duchenne Muscular Dystrophy

被引：4

作者：

Yoshioka, Kantaro ^{[1
]}

Ito, Akira ^{[2
]}

Horie, Masanobu ^{[3
]}

Ikeda, Kazushi ^{[4
]}

Kataoka, Sho ^{[1
]}

Sato, Keiichiro ^{[1
]}

Yoshigai, Taichi ^{[1
]}

Sakurai, Hidetoshi ^{[5
]}

Hotta, Akitsu ^{[5
]}

Kawabe, Yoshinori ^{[1
]}

Kamihira, Masamichi ^{[1
,4
]}

机构：

[1] Kyushu Univ, Fac Engn, Dept Chem Engn, Fukuoka 8190395, Japan

[2] Nagoya Univ, Sch Engn, Dept Chem Syst Engn, Nagoya 4648603, Japan

[3] Kyoto Univ, Radioisotope Res Ctr, Div Biochem Engn, Kyoto 6068501, Japan

[4] Kyushu Univ, Grad Sch Syst Life Sci, Fukuoka 8190395, Japan

[5] Kyoto Univ, Ctr iPS Cell Res & Applicat, Kyoto 6068507, Japan

来源：

CELLS | 2021年 / 10卷 / 10期

基金：

日本学术振兴会;

关键词：

Duchenne muscular dystrophy; human induced pluripotent stem cell; myotube; contractile activity; CRISPR/Cas9; MDX; DECORIN; GENE;

D O I：

10.3390/cells10102556

中图分类号：

Q2 [细胞生物学];

学科分类号：

071009 ; 090102 ;

摘要：

Duchenne muscular dystrophy (DMD) is a genetic disorder that results from deficiency of the dystrophin protein. In recent years, DMD pathological models have been created using induced pluripotent stem (iPS) cells derived from DMD patients. In addition, gene therapy using CRISPR-Cas9 technology to repair the dystrophin gene has been proposed as a new treatment method for DMD. However, it is not known whether the contractile function of myotubes derived from gene-repaired iPS cells can be restored. We therefore investigated the maturation of myotubes in electrical pulse stimulation culture and examined the effect of gene repair by observing the contractile behaviour of myotubes. The contraction activity of myotubes derived from dystrophin-gene repaired iPS cells was improved by electrical pulse stimulation culture. The iPS cell method used in this study for evaluating muscle contractile activity is a useful technique for analysing the mechanism of hereditary muscular disease pathogenesis and for evaluating the efficacy of new drugs and gene therapy.

引用

页数：13

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