EXPRESSION OF DYSTROPHIN AND ITS ASSOCIATED PROTEINS FOLLWING MYOBLAST TRANSPLANTATION INTO MDX MICE.

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Dystropin is a responsible protein of Duchenne muscular dystrophy (DMD) and is present at the protoplasmic surface of normal sarcolemma. Its absencd is observed in DMD and mdx (mouse equivalent to DMD) muscles and is considered to be the cause of muscle fiber degeneration. To construct the subsarcolemmal dystrophin-undercoat in mdx muscle, myoblasts with normal dystrophy gene are injected into mdx mouse muscle. We injected C2 myblasts, a normal mouse muscle cell line, into the muscles of mdx nude mouse. A large number of dystrophin-positive fibers appeared after C2 myoblast transplantation. We examined dystrophin-positive fibers with antidystrophin associated proteins antibodies. We reported that dystrophin-associated proteins are biochemically classified into 3 groups, namely, dystroglycan complex and syntrophin complex. We also cloned a new sarcoglycan (gamma-sarcoglydan) cDNA and showed that the human gamma-sarcoglycan gene was mappetd to chromosome 13q12 and delations that alter its reading frame were identified in three families and one of four sporadic cases of SCARMD.We showed the efficient transduction and expression of lacZ gene in the skeletal muscle of adult C57BL/10ScSn mice after adenovirus-mediated gene transfer. Sixty-two percent of the myofibers in the tibialis anterior (TA) muscle were b-galactosidase-positive after injection of the lacZ gene under the control of the chicken b-actin promoter and the cytomegalovirus enhancer. The transduced gene was preferentially expressed in type IIA and IIX fibers which were richer in oxidative enzymes than type IIB fibers.