255. Embryonic Stem Cell-Mediated Regenerative Therapy for Duchenne Muscular Dystrophy

Introduction: Duchenne muscular dystrophy (DMD) is a severe muscle degenerative disorder caused by mutations in the dystrophin gene. Embryonic stem cell (ES cell) transplantation is one of the more promising therapies because ES cells are available in large quantities and can serve to systemically restore affected muscles of DMD patients. Although ES cells have shown to be capable of differentiating into various tissues and cell types, it has been difficult to induce differentiation of ES cells specifically into muscle fibers only. We are proposing a method to overcome this impediment by establishing genetically engineered ES cells which harbor a tetracycline-regulated expression vector encoding the myogenic transcriptional factor MyoD.