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

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… (More)
DOI: 10.1007/978-1-61737-982-6_2

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Cite this paper

@article{Athanasopoulos2011CodonOO, title={Codon optimization of the microdystrophin gene for Duchene muscular dystrophy gene therapy.}, author={Takis Athanasopoulos and Helen A. Foster and Keith Foster and George Dickson}, journal={Methods in molecular biology}, year={2011}, volume={709}, pages={21-37} }