Foundations for cell therapy in muscle disorders. GMP considerations for clinical cell products. Commercialization and investment issues. Regulatory issues for cell therapy. Immunologic considerations for cell transplantation. Developmental biology and muscle cell transplantation. Identification and Characterization of Satellite Stem Cells. Hemangioblasts in skeletal muscle disorders.
Derivation of muscle cells from iPS. Primary muscles cells and transplantation. Use of physical modalities in muscle cell transplantation. Use of viral vectors for ex vivo correction of muscle cells. Role of animal models in cell therapy for muscle disorders. Specific Disorders. Genetic correction of MPCS derived from iPS cells for LGMD. Myoblast transplantation in DMD.
Genetic correction of human iPS for transplantation in spinal muscular atrophy. Local injection of stem cells in the diaphragm for DMD. In utero and Neonatal Stem Cell Therapy for Duchenne Muscular Dystrophy. Mechanically Programmed Adipose-Derived Stem Cells to Treat Muscular Dystrophy.