REVERSIBLE IMMORTALIZATION AND TRANSDUCTION WITH A DYSTROPHIN ARTIFICIAL CHROMOSOME OF HUMAN DMD MESOANGIOBLASTS FOR THE CELL THERAPY OF MUSCULAR DYSTROPHY

We plan to develop a new strategy that will allow to "cure" the patient stem cells in vitro, by transferring the whole dystrophin gene inside an artificial chromosome. In order to achieve this goal the cells will have to be expanded in vitro for a large number of cell divisions (because the transfer of the chromosome is inefficient and only occurs in very few cells). This will be achieved by reversibly immortalizing stem cells with telomerase gene that contrasts cell senescence. Before transplantation into dystrophic, immune deficient mice, these immortalizing genes will be removed from the cells. A large number of safety control, will guarantee that these modified cells may not give rise to tumors, thus resulting in an efficacious and safe novel tool to treat muscular dystrophy.