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

  • Takis Athanasopoulos
  • Helen Foster
  • Keith Foster
  • George Dickson
Protocol
Part of the Methods in Molecular Biology book series (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.

Key words

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

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Copyright information

© Springer Science+Business Media, LLC 2011

Authors and Affiliations

  • Takis Athanasopoulos
    • 3
  • Helen Foster
    • 1
    • 2
  • Keith Foster
    • 1
    • 2
  • George Dickson
    • 1
    • 2
    • 3
  1. 1.Institute of Biomedical and Life Sciences, South West London Academic NetworkSt. George’s University of LondonLondonUK
  2. 2.Centre for Biomedical Sciences, School of Biological Sciences, Royal HollowayUniversity of LondonLondonUK
  3. 3.School of Biological Sciences, Royal HollowayUniversity of LondonLondonUK

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