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Codon Optimization of the Microdystrophin Gene for Duchenne Muscular Dystrophy Gene Therapy

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Muscle Gene Therapy

Part of the book series: Methods in Molecular Biology ((MIMB,volume 709))

Abstract

Duchenne muscular dystrophy (DMD) is a severe muscle wasting X-linked genetic disease caused by dystrophin gene mutations. Gene replacement therapy aims to transfer a functional full-length dystrophin cDNA or a quasi micro/mini-gene into the muscle. A number of AAV vectors carrying microdystrophin genes have been tested in the mdx model of DMD. Further modification/optimization of these microgene vectors may improve the therapeutic potency. In this chapter, we describe a species-specific, codon optimization protocol to improve microdystrophin gene therapy in the mdx model.

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References

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Author information

Authors and Affiliations

  1. Institute of Biomedical and Life Sciences, South West London Academic Network, St. George’s University of London, London, UK

    Helen Foster, Keith Foster & George Dickson

  2. Centre for Biomedical Sciences, School of Biological Sciences, Royal Holloway, University of London, Egham, London, UK

    Helen Foster, Keith Foster & George Dickson

  3. School of Biological Sciences, Royal Holloway, University of London, Egham, London, UK

    Takis Athanasopoulos & George Dickson

Authors
  1. Takis Athanasopoulos
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  2. Helen Foster
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  3. Keith Foster
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  4. George Dickson
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Corresponding author

Correspondence to Takis Athanasopoulos .

Editor information

Editors and Affiliations

  1. , Dept. of Molecular Microbiology, University of Missouri, Hospital Dr. 1, Columbia, 65212, Missouri, USA

    Dongsheng Duan

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Athanasopoulos, T., Foster, H., Foster, K., Dickson, G. (2011). Codon Optimization of the Microdystrophin Gene for Duchenne Muscular Dystrophy Gene Therapy. In: Duan, D. (eds) Muscle Gene Therapy. Methods in Molecular Biology, vol 709. Humana Press. https://doi.org/10.1007/978-1-61737-982-6_2

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  • DOI: https://doi.org/10.1007/978-1-61737-982-6_2

  • Published:

  • Publisher Name: Humana Press

  • Print ISBN: 978-1-61737-981-9

  • Online ISBN: 978-1-61737-982-6

  • eBook Packages: Springer Protocols

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