Protocol

Muscle Gene Therapy

Volume 709 of the series Methods in Molecular Biology pp 21-37

Date:

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

  • Takis AthanasopoulosAffiliated withSchool of Biological Sciences, Royal Holloway, University of London Email author 
  • , Helen FosterAffiliated withInstitute of Biomedical and Life Sciences, South West London Academic Network, St. George’s University of LondonCentre for Biomedical Sciences, School of Biological Sciences, Royal Holloway, University of London
  • , Keith FosterAffiliated withInstitute of Biomedical and Life Sciences, South West London Academic Network, St. George’s University of LondonCentre for Biomedical Sciences, School of Biological Sciences, Royal Holloway, University of London
  • , George DicksonAffiliated withInstitute of Biomedical and Life Sciences, South West London Academic Network, St. George’s University of LondonCentre for Biomedical Sciences, School of Biological Sciences, Royal Holloway, University of LondonSchool of Biological Sciences, Royal Holloway, University of London

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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.

Key words

Codon optimization mRNA AAV Adeno-associated virus Duchenne muscular ­dystrophy Dystrophin Microdystrophin Minidystrophin Quasidystrophin Gene therapy Muscle mdx