CELL THERAPY OF MUSCULAR DYSTROPHY WITH ENGINEERED CD133+ STEM CELLS

Aim of this project is to develop a novel approach for the therapy of the neuromuscular diseases based on the combination of the stem cell delivery and a new gene therapy technology named exon skipping. By this approach we will move to provide a rationale for the autologous transplantation. We will establish strategies based on cell therapy tissue repair by the contribution of genetically modified stem cells repairing the defective gene. This approach would permit the use of the patients own cells (autologous transplantation) reducing the risk of donor implant rejection. The aims are the follows: characterization, isolation and expansion of a subpopulation of human stem cells isolated from muscle and blood tissues of healthy donors and dystrophic patients; genetic engineering of human dystrophic stem cells by exon skipping approach injection of engineered dystrophic stem cells in animals models of muscular dystrophies such as the scid/mdx mice and the golden retriever (GRMD) dog (both animal models of Duchenne Muscular Dystrophy, DMD), analyse of their potentiality to regenerate muscle fibres; evaluation of the safety of this protocol for future potential clinical use.