PARTIAL RESTORATION OF DYSTROPHIN IN MDX MICE FOLLOWING SYSTEMIC ADMINISTRATION OF GENETICALLY ENGINEERED MUSCLE DERIVED STEM CELLS

@inproceedings{Petersen2001PARTIALRO,
  title={PARTIAL RESTORATION OF DYSTROPHIN IN MDX MICE FOLLOWING SYSTEMIC ADMINISTRATION OF GENETICALLY ENGINEERED MUSCLE DERIVED STEM CELLS},
  author={Qu Petersen and Jankowski and Pruchnic and Kimura and Ikezawa and James D. Cummins and Huard},
  year={2001}
}
Introduction: Duchenne Muscular Dystrophy (DMD) is a muscle disease characterized by a lack of dystrophin expression resulting in severe muscle weakness and early death due to cardiac and respiratory failures. Various approaches have been used to deliver dystrophin to the diseased muscle including myoblast transplantation (MT), gene therapy (GT), and ex vivo approach. The poor survival and the limited spread of transplanted myoblasts are two majors hurdles facing the overall application of MT… CONTINUE READING