Human dystrophin expression in mdx mice after intramuscular injection of dna constructs

摘要: DUCHENNE'S muscular dystrophy (DMD), which affects one in 3,500 males, causes progressive myopathy of skeletal and cardiac muscles premature death1. One approach to treatment would be introduce the normal dystrophin gene into diseased muscle cells. When pure plasmid DNA is injected rodent skeletal2 or muscle3–5, cells express reporter genes. We now show that a 12-kilobase full-length human complemen-tary 6.3-kilobase Becker-like gene6 can expressed cultured vivo. expression plasmids are intramuscularly dystrophin-deficient mdx mice, proteins present cytoplasm sarcolemma ∼1% myofibres. Myofibres expressing contain an increased proportion peripheral nuclei. The results indicate transfer myofibres DMD patients could beneficial, but larger number genetically modified will necessary for clinical efficacy.