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

Methods Mol Biol. 2011:709:21-37. doi: 10.1007/978-1-61737-982-6_2.

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.

MeSH terms

  • Animals
  • Base Sequence
  • Codon*
  • DNA, Complementary / genetics
  • Dependovirus / genetics
  • Dystrophin / genetics*
  • Genetic Therapy / methods*
  • Genetic Vectors
  • Humans
  • Mice
  • Mice, Inbred mdx
  • Muscles
  • Muscular Dystrophy, Animal / therapy
  • Muscular Dystrophy, Duchenne / genetics
  • Muscular Dystrophy, Duchenne / therapy*

Substances

  • Codon
  • DNA, Complementary
  • Dystrophin