ADIPOSE TISSUE-DERIVED MYOGENIC PREURSORS FOR THE TREATMENT OF MUSCULAR DYSTROPHY

Duchenne Muscular Dystrophy (DMD) is a progressive muscle wasting disease affecting 1 in 3500 boys. Patients are usually confined to wheelchair before age of 12 and die their late teens or early twenties for respiratory and heart failure. DMD is caused by a mutation in a gene coding for a protein, called Dystrophin, which is specifically expressed in muscle cells. Among various therapeutical approaches for the treatment of DMD, cell therapy, in particular, is based on the transplantation or injection of stem cells expressing the correct copy of Dystrophin, which are able to form muscle cells and which, by fusion with host muscle fibers, can regenerate the diseased tissue. It has actually been shown that stem cells of different origin may restore dystrophin expression in muscles and heart of animal models of muscular dystrophy. However, also due to a reduced availability of myogenic stem cells in DMD patients, further studies are necessary before cell therapy may be effectively used for the treatment of DMD. Research studies in our laboratory led to the discovery of myogenic precursor cells in the adipose tissue. Compared to other sources of stem cells such as bone marrow, blood, or the muscle itself, adipose tissue offer the advantage to be highly available and accessible. Aim of our project is to characterize the potentiality of adipose tissue as a new source of myogenic progenitors to be used for the cell therapy of muscular dystrophy.