Article
Adenovirus-based targeting in myoblasts is hampered by nonhomologous vector integration.
Human gene therapy - 1 Oct 2008
Isman Olga, Roberts Michael L, Morgan Jennifer E, Graham Ian R, Goldring Kirstin, Lawrence-Watt Diana J, Lu Qi Long, Dunckley Matthew G, Porter Andrew C G, Partridge Terence A, Dickson George
Abstract excerpt
Chromosomal correction of dystrophin gene mutations is a most desirable therapeutic solution for Duchenne muscular dystrophy, as it allows production of the full-length dystrophin under the control of locus-specific promoters. Here we explored gene targeting in conditionally immortal mouse dystrophin-deficient myoblasts. We constructed an adenoviral vector for the correction of the mdx mutation, containing 6.0 kb...
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