Contractile Activity of Myotubes Derived from Human Induced Pluripotent Stem Cells: A Model of Duchenne Muscular Dystrophy.
Kantaro YoshiokaAkira ItoMasanobu HorieKazushi IkedaSho KataokaKeiichiro SatoTaichi YoshigaiHidetoshi SakuraiAkitsu HottaYoshinori KawabeMasamichi KamihiraPublished in: Cells (2021)
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.
Keyphrases
- duchenne muscular dystrophy
- gene therapy
- induced apoptosis
- skeletal muscle
- muscular dystrophy
- genome wide
- copy number
- cell cycle arrest
- induced pluripotent stem cells
- crispr cas
- smooth muscle
- endothelial cells
- blood pressure
- ejection fraction
- endoplasmic reticulum stress
- cell death
- signaling pathway
- high glucose
- single cell
- resistance training
- transcription factor
- pi k akt
- patient reported outcomes
- high resolution
- cell proliferation
- gene expression
- smoking cessation
- diabetic rats
- high intensity
- bone marrow
- atomic force microscopy
- combination therapy
- binding protein