EXPERIMENTAL CHALLENGE FOR THE TREATMENT OF DUCHENNE MUSCULAR-DYSTROPHY USING A VASCULARIZED FREE MUSCLE GRAFT

Following free vascularized normal muscle graft in mice, a study was m ade to determine whether dystrophin expression is possible in dystroph in-deficient muscles. In this study, dystrophic C57BL/10 ScSn-mdx mice were used as recipients and normal C57BL/10 ScSn mice as donors. A fr ee vascularized quadriceps muscle 8.0 x 6.0 x 6.0 mm in size was ortho topically transplanted into a muscle defect produced in the recipient mouse. The diameter of the sutured vessels was about 0.4 mm. Transplan tation was successful in 7 of 20 mice. At 12 weeks after the transplan tation, the grafted muscle was examined by immunocytochemical stain us ing anti-dystrophin antibody. This study showed that dystrophin was ex pressed in the transplanted muscle but not in the adjacent recipient q uadriceps muscle, suggesting that grafted donor cells with dystrophin failed to migrate into dystrophic muscle cells and fuse together. Howe ver, since the grafted normal skeletal muscle successfully survived an d normal dystrophin was expressed in almost all the grafted muscle fib ers, the possibility was suggested that the function of muscular dystr ophy muscle can be compensated by complete replacement with a larger m uscle. (C) 1994 Wiley-Liss, Inc.