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Duchenne muscular dystrophy (DMD) is a fatal progressive muscle wasting disease caused by defects in the dystrophin gene. No viral vector except the helper-dependent adenovirus vector (HDAdv) can package 14kb full-length dystrophin cDNA and HDAdv is considerably safer than old-generation adenovirus vectors due to the large-size deletion in its genome. I have generated HDAdv that carries myc-tagged murine full-length dystrophin cDNA (HDAdv-myc-mFLdys). I injected it into the multiple proximal muscles of 7-day-old utrophin/dystrophin double knockout mice (dko mice), which typically show symptoms quite similar to human DMD, because the proximal muscles are organs affected in DMD patients. Eight weeks after injections, the transduced dystrophin was widely expressed and I found a significant reduction ofcentrally nucleated myofibers and the restoration of dystrophin associated proteins, β-dystroglycan (β-DG) and α-sarcoglycan (α-SG), as well as neuronal nitric oxide synthase(nNOS). The injected dko mice also showed an increase in body weight, an improvement in motor performances, and prolonged lifespan. Using HDAdv, I couldtreat DMD model mice, even when the therapeutic gene was transferred into multiple skeletal muscles. These results suggest that multiple intramuscular administrations of HDAdv carrying full-length dystrophin may reduce symptoms and compensate for lost functions in DMD patients., 本研究では、dkoマウスの四肢近位筋および体幹の骨格筋にHDAdvを用いて完全長dystrophinを導入し、その治療効果を病理学的観点および運動機能の観点から評価、検討する。