Addressing Cell Therapy for Spinal Muscular Atrophy: Open Issues and Future Perspectives

Spinal muscular atrophy (SMA) is a devastating neuromuscular disease that affects children and young adults. The degeneration of spinal motor neurons, which are primarily involved in this disease, results in progressive muscular weakness and atrophy. As of 2016, there is no effective clinical treatment available for SMA patients, even if a large number of therapeutic approaches are being tested worldwide.Stem cells have been considered as a potential therapeutic strategy due to their plasticity and ability to direct differentiation in response to extracellular signals. Current cell-based therapies employ multiple types of stem cells to modify disease pathophysiology. This is theoretically achieved by either supporting neurons and glial cells through the release of neurotrophic factors, or directly replacing them.This chapter focuses on the therapeutic potential of different subtypes of human pluripotent stem cells and their derived phenotypes for SMA. This chapter also briefly describes their contribution in disease pathogenesis understanding. Finally, the chapter discusses the main challenges to overcome for stem cell therapeutic clinical translation.