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Genetics

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JoVE Journal Genetics
Exon Overslaan in direct geherprogrammeerd Myotubes verkregen uit menselijke urine-afgeleide cellen
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Exon Overslaan in direct geherprogrammeerd Myotubes verkregen uit menselijke urine-afgeleide cellen

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 dit artikel beschrijven we een gedetailleerd protocol voor het efficiënt modelleren van Duchenne spierdystrofie met behulp van MYOD1-geconverteerdeurine-afgeleide cellen om het herstel van dystrofine mRNA en eiwitniveaus te evalueren na exon overslaan.

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Genetica Duchenne spierdystrofie (DMD) urine-afgeleide cellen exon overslaan precisiegeneeskunde skeletspier klinische proef
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