Cell therapy for myopathies


Group leader

Giulio Cossu


Cell therapy for myopathies laboratory has a long-lasting interest and more than 40 years of research experience in the feld of muscle biology and muscular dystrophy. We identified signals that activate myogenesis in the embryo, discovered the myogenic potential of bone-marrow derived, circulating progenitor cells, identified of a novel population of vessel associated progenitor cells that we termed mesoangioblasts, later characterized as a subset of muscle pericytes. We tested the therapeutic potential of mesoangioblasts in mouse and dog models of muscular dystrophy and completed a phase I/II clinical trial in five Duchenne muscular dystrophy patients who received escalating doses of HLA-matched, donor derived mesoangioblasts.

Research activity

We are currently optimising the transplantation protocol and have started a new trial with autologous, genetically corrected cells. The juxtaposition of developmental biology with stem cell clinical work characterises our work in the field of molecular medicine.