Helper-dependent adenovirusvector and modified lentiviral vector mediated delivery of dystrophin for gene therapy of muscular dystrophy

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We showed that a highly efficient dystrophin-transduction to the mex and dko's diaphragm-achieved by simple intraperitoneal injection of a helper-dependent adenovirus vector (HDAdv) containing the full-length dystrophin expression cassette - provided beneficial results. we carried out the packaging of the full-length dystrophin cDNA in a genetically modified lentivirus with a useful, sufficiently functional titer that was successfully transduced into myogenic cells derived from the transgenic mdx mouse.