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Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy

DOI :

10.3791/53776-v

10:30 min

May 24th, 2016

May 24th, 2016

18,179 Views

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

Exon skipping is currently a most promising therapeutic option for Duchenne muscular dystrophy (DMD). To expand the applicability for DMD patients and to optimize the stability/function of the resulting truncated dystrophin proteins, a multi-exon skipping approach using cocktail antisense oligonucleotides was developed and we demonstrated systemic dystrophin rescue in a dog model.

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Multi exon Skipping

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