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Translational Research in Nucleic Acid Therapies for Muscular Dystrophies

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Translational Research in Muscular Dystrophy

Abstract

Nucleic acid therapies have gained significant traction in recent years as a promising new approach to treating various genetic diseases. Such therapies employ synthetic, small molecules called antisense oligonucleotides (AOs) which are capable of modulating the transfer of genetic information from nucleic acid to protein through various mechanisms, including the augmentation of pre-mRNA splicing and downregulation of expression. Thus, AOs can prevent the incorporation of genetic mutations causing disease into final protein transcripts as well as reduce levels of mutant transcripts, potentially ameliorating disease phenotype. This process, also known as antisense therapy, has recently been the subject of several preclinical and clinical trials aimed at treating muscular dystrophies. Thanks to recent advancements in antisense drug chemistries, numerous studies have demonstrated the safety, tolerability, and efficacy of AOs administered to patients with Duchenne muscular dystrophy, the most common form of muscular dystrophy. In the wake of promising clinical trial data, it may well be that the first federally approved marketable antisense drug for treating muscular dystrophy could be on the horizon.

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Acknowledgments

This work was supported by the University of Alberta Faculty of Medicine and Dentistry, Parent Project Muscular Dystrophy (USA), The Friends of Garrett Cumming Research Funds, HM Toupin Neurological Science Research Funds, Muscular Dystrophy Canada, Canada Foundation for Innovation, Alberta Enterprise and Advanced Education, Jesse’s Journey, Slipchuk SMA Research Funds, the Women and Children’s Health Research Institute, and Canadian Institutes of Health Research.

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  1. Department of Medical Genetics, University of Alberta Faculty of Medicine and Dentistry, 8812-112 Street, Edmonton, AB, T6G 2H7, Canada

    Joshua J. A. Lee & Toshifumi Yokota

  2. Muscular Dystrophy Canada Research Chair, Edmonton, AB, Canada

    Toshifumi Yokota

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  1. Joshua J. A. Lee
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  2. Toshifumi Yokota
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Correspondence to Toshifumi Yokota .

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Editors and Affiliations

  1. National Institute of Neuroscience, Kodaira, Tokyo, Tokyo, Japan

    Shin'ichi Takeda

  2. National Institute of Neuroscience, Kodaira, Tokyo, Tokyo, Japan

    Yuko Miyagoe-Suzuki

  3. National Institute of Neuroscience, Kodaira, Tokyo, Tokyo, Japan

    Madoka Mori-Yoshimura

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Lee, J.J.A., Yokota, T. (2016). Translational Research in Nucleic Acid Therapies for Muscular Dystrophies. In: Takeda, S., Miyagoe-Suzuki, Y., Mori-Yoshimura, M. (eds) Translational Research in Muscular Dystrophy. Springer, Tokyo. https://doi.org/10.1007/978-4-431-55678-7_6

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  • DOI: https://doi.org/10.1007/978-4-431-55678-7_6

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  • Publisher Name: Springer, Tokyo

  • Print ISBN: 978-4-431-55677-0

  • Online ISBN: 978-4-431-55678-7

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