title: [Stem cell based cell therapy for muscular dystrophy].
abstract: Stem cell-based cell therapy is one of attractive therapeutic approaches to muscular dystrophy. In transplantation into dystrophic skeletal muscle, muscle satellite cells and musclE side population (SP) cells are good candidates as sources of stem cells. We purified mouse satellite cells from adult C57B16 mice by FACS using a monoclonal antibody, SM/C-2.6, which can specifically recognize quiescent satellite cells. DNA micro-array analysis on both quiescent and activated satellite cells allowed us to distinguish novel quiescent satellite cell-specific genes. These genes may encode molecules that keep satellite cells in a dormant and undifferentiated state. We transplanted purified muscle satellite cells transduced with lentivirus vector, and found these cells were effectively incorporated into dystrophin-deficient skeletal muscle and expressed transduced dystrophin. We also identified a novel side population (SP) cells in uninjured and regenerating skeletal muscle. The majority of muscle-SP cells in uninjured stage showed endothelial cell-like properties. CD31 negative/CD45 negative SP cells were a minor population in normal conditions, but actively proliferate during muscle regeneration. Co-transplantation experiments showed that the SP cells synergistically promoted muscle regeneration with satellite cells. It is indispensable to study molecular basis of muscle stem cells and muscle regeneration to achieve effective stem cell-based cell therapy on muscular dystrophy.
authors: Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry.
authors: Shin'ichi
authors: S
authors: Takeda
mesh_terms: D000818:Animals; D051379:Mice; D008807:Mice, Inbred BALB C; D009137:Muscular Dystrophy, Animal / Q000628:therapy*; D033581:Stem Cell Transplantation / Q000379:methods*