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

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

6,741 Views

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06:20 min

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May 07, 2020

DOI:

10.3791/60840-v

DOI:
10.3791/60840-v

•

06:20 min
May 07, 2020

•

2 Views
, ,
1Department of Molecular Therapy, National Institute of Neuroscience,National Center of Neurology and Psychiatry

Summary

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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 SkippingDirect ReprogrammingHuman Urine-derived CellsMyotubesDuchenne Muscular Dystrophy3-deazaneplanocin A HydrochlorideDZnepHistone Methyltransferase InhibitorAutologousPrecision MedicineMuscular DiseasesCentrifugationCell CultureGelatin-coatedRetrovirusMYOD1Puromycin

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Takizawa, H., Sato, M., Aoki, Y. Exon Skipping in Directly Reprogrammed Myotubes Obtained from Human Urine-Derived Cells. J. Vis. Exp. (159), e60840, doi:10.3791/60840 (2020).

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