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Journal / Medicine / Multi-exon Skipping Met behulp Cocktail…
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Multi-exon Skipping Met behulp Cocktail antisense-oligonucleotiden in de Canine X-gebonden Muscular Dystrophy
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Multi-exon Skipping Met behulp Cocktail antisense-oligonucleotiden in de Canine X-gebonden Muscular Dystrophy

Article DOI: 10.3791/53776-v • 10:30 min • May 24th, 2016
May 24th, 2016 •
*1, *2, 2, 1, 2, 1
1Department of Medical Genetics, University of Alberta Faculty of Medicine and Dentistry, 2Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry
* These authors contributed equally

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Exon skipping is momenteel een veelbelovende therapeutische optie voor Duchenne spierdystrofie (DMD). Om de toepasbaarheid voor DMD patiënten uit te breiden en de stabiliteit / functie van de resulterende afgeknotte dystrofine-eiwit te optimaliseren, een multi-exon skipping benadering met behulp van cocktail antisense oligonucleotiden werd ontwikkeld en we aangetoond systemische dystrofine redding bij een hond model.

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Geneeskunde Duchenne spierdystrofie (DMD) Canine X-gebonden spierdystrofie (CXMD) spierbiopsie fosfordiamidaat morfolino-oligomeren (morpholinos of PMO) Dystrofine 2'O-methyl antisense oligonucleotiden (2'OMePS) Skeletal spier Octa-guanidine dendrimeer geconjugeerd morpholinos (vivo-morpholinos of vPMOs) mRNA splicing exon splice enhancer (ESE) Becker spierdystrofie (BMD) antisense oligonucleotide (AON) gemedieerde exon skipping therapie
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