title: 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 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.
authors.affiliation: Institute of Biomedical and Life Sciences, South West London Academic Network, St. George's University of London, London, UK. P.Athanasopoulos@rhul.ac.uk
authors.forename: Takis
authors.initials: T
authors.lastname: Athanasopoulos
authors.forename: Helen
authors.initials: H
authors.lastname: Foster
authors.forename: Keith
authors.initials: K
authors.lastname: Foster
authors.forename: George
authors.initials: G
authors.lastname: Dickson
mesh_terms: D000818:Animals; D001483:Base Sequence; D003062:Codon; D018076:DNA, Complementary / Q000235:genetics; D000229:Dependovirus / Q000235:genetics; D016189:Dystrophin / Q000235:genetics*; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006801:Humans; D051379:Mice; D018101:Mice, Inbred mdx; D009132:Muscles; D009137:Muscular Dystrophy, Animal / Q000628:therapy; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics / Q000628:therapy*
chemical_list: D003062:Codon; D018076:DNA, Complementary; D016189:Dystrophin