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Journal / Genetics / Exon Skipping in Directly Reprogrammed Myotubes…
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Genetics

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JoVE Journal Genetics
Exon Skipping in Directly Reprogrammed Myotubes Obtained from Human Urine-Derived Cells
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Exon Skipping in Directly Reprogrammed Myotubes Obtained from Human Urine-Derived Cells

Article DOI: 10.3791/60840-v • 06:20 min • May 7th, 2020
May 7th, 2020 •
1, 1, 1
1Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry

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In this article, we describe a detailed protocol for efficient modelling of Duchenne muscular dystrophy muscle using MYOD1-converted urine-derived cells to evaluate the restoration of dystrophin mRNA and protein levels after exon skipping.

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Exon Skipping Directly Reprogrammed Myotubes Human Urine-derived Cells In Vitro Modeling Duchenne Muscular Dystrophy 3-deazaneplanocin A Hydrochloride Histone Methyltransferase Inhibitor UDC-based Disease Modeling Precision Medicine Centrifuge Supernatant Pellets Washing Buffer Primary Medium Gelatine-coated Six-well Plate Cell Suspension Growth Medium
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