• Methods Mol. Biol. · Jan 2011

    Codon optimization of the microdystrophin gene for Duchene muscular dystrophy gene therapy.

    • Takis Athanasopoulos, Helen Foster, Keith Foster, and George Dickson.
    • Institute of Biomedical and Life Sciences, South West London Academic Network, St. George's University of London, London, UK. P.Athanasopoulos@rhul.ac.uk
    • Methods Mol. Biol. 2011 Jan 1; 709: 21-37.

    AbstractDuchenne 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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