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3 articles published in JoVE
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Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy
Bailey Miskew Nichols*1, Yoshitsugu Aoki*2, Mutsuki Kuraoka2, Joshua J.A. Lee1, Shin'ichi Takeda2, Toshifumi Yokota1
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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