Cell replacement therapy in neuromuscular and neurodegenerative diseases

In the past 10 years there have been important advances in our understanding of the pathogenesis and pathophysiology of inherited and acquired neuromuscular and neurodegenerative diseases. Cell-based therapies were used to promote muscle regeneration with the hope that the host cells repopulated the muscle and improved muscle function and pathology. Stem cells were preferable for therapeutic applications, due to their capacity for self-renewal and differentiative potential. Adult stem cells were found in various tissues of the body and they were able to maintain, generate, and replace terminally differentiated cells within their own specific tissue because of cell turnover or tissue injury. It became clear that these cells could participate in regeneration of more than just their resident organ. In the last years, encouraging results were obtained by utilizing stem cells with degenerative diseases such as Parkinson’s disease, Motor Neuron Disease, Multiple Sclerosis, Amyotrophic Lateral Sclerosis and Alzheimer’s Disease. Moreover, different works demonstrated how myogenic and non-myogenic stem cells could exert a significant role in the treatment of muscular dystrophies. The knowledge of stem cells coupled with novel stem cell-based delivery systems, and advances in modulation of immune mechanisms, modification of the activity of mutant genes, and performing gene replacement therapies will expand our ability to treat neuromuscular and neurodegenerative diseases.