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  • Title: Codon optimization of the microdystrophin gene for Duchene muscular dystrophy gene therapy.
    Author: Athanasopoulos T, Foster H, Foster K, Dickson G.
    Journal: Methods Mol Biol; 2011; 709():21-37. PubMed ID: 21194019.
    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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