Restoration of muscle fibers and satellite cells after isogenic MSC transplantation with microdystrophin gene delivery.
Duchenne muscular dystrophy is the most prevalent inheritable muscle disease. Transplantation of autologous stem cells with gene direction is an ideal therapeutic approach for the disease. The current study aimed to investigate the restoration of myofibers in mdx mice after mdx bone marrow-derived mesenchymal stem cell (mMSC) transplantation with human microdystrophin delivery. Possible mechanisms of action were also studied. In our research, mMSCs were successfully transduced by retrovirus carrying a functional human microdystrophin gene. Transplantation of transduced mMSCs enabled persistent dystrophin restoration in the skeletal muscle of mdx mice up to the 12th week after transplantation. Simultaneous coexpression of human microdystrophin and desmin showed that implanted mMSCs are capable of long-term survival as muscle satellite cells.
Department of Neurology, First Affiliated Hospital, Sun Yat-sen University, Guangzhou, PR China; Institute of Population Research, Peking University, PR China.
Shan-wei
SW
Feng
Fei
F
Chen
Jiqing
J
Cao
Mei-juan
MJ
Yu
Ying-yin
YY
Liang
Xin-ming
XM
Song
Cheng
C
Zhang
D000818:Animals; D004195:Disease Models, Animal; D016189:Dystrophin / Q000235:genetics*; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D045164:Mesenchymal Stem Cell Transplantation / Q000379:methods*; D051379:Mice; D018485:Muscle Fibers, Skeletal / Q000378:metabolism / Q000502:physiology*; D020388:Muscular Dystrophy, Duchenne / Q000628:therapy*; D012190:Retroviridae; D032496:Satellite Cells, Skeletal Muscle / Q000378:metabolism / Q000502:physiology*
D016189:Dystrophin