Muscular dystrophies are characterized by severe muscle damage, ultimately through the loss of the ability to regenerate muscle. Generating alternative sources for precursor cells to replenish muscle fibers offers a potential therapeutic strategy to treat myopathies. Yvan Torrente and colleagues, from the University of Milan, having previously shown that a cellular marker called AC133 is required for muscle precursor cells to differentiate into mature muscle, the researchers now demonstrate that AC133-positive stem cells are a promising new source for replenishing muscle satellite cells that are depleted in muscular dystrophies. The researchers isolated human AC133-positive stem cells from normal blood. After growth in culture, these cells were subsequently injected into the skeletal muscle tissue of mice that are a model for human Duchene muscular dystrophy. The human stem cells invaded the damaged muscle fibers, expressed muscle fiber markers, and formed functional myofibers that restored muscle function. The injected mice also experienced amelioration of the clinical symptoms of muscular dystrophy and a restoration of the satellite cell pool required for muscle regeneration. This study provides new leads in the treatment of these debilitating muscle diseases.
TITLE: Human circulating AC133+ stem cells restore dystrophin expression and ameliorate function in dystrophic skeletal muscle
Yvan Torrente University of Milan, Padiglione Ponti, Ospedale Policlinico, via Francesco Sforza 35, 20122 Milan, Italy.
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