[Introduction of rod-deleted dystrophin cDNA, delta DysM3, into mdx skeletal muscle using adenovirus vector]

The 6.3 kb mini-dystrophin cDNA, has been successfully introduced in dystrophic mdx mouse and phenotypes of muscular dystrophy has been considerably improved. We generate a dystrophin cDNA construct deleted more for rod domain of minidystrophin, resulting only one rod repeat with two hinge segments, 3.7 kb delta DysM3. Recombinant adenovirus vector, Ax delta DysM3 has been made using COS-TPC method. 125 kDa delta DysM3 was expressed in the cultured skeletal muscle cells, when Ax delta DysM3 was infected. delta DysM3 can effectively accumulate in sarcolemma and considerably recover of dystrophin-associated proteins when transferred into skeletal muscle of mdx mouse. This small-sized rod-deleted dystrophin can be incorporated into adeno-associated virus vector, which was recently proven to be good tool to carry the gene into skeletal muscle.