Perspectives of stem cell therapy in Duchenne muscular dystrophy

• Published in: The FEBS journal 2013-09, Vol.280 (17), p.4251-4262
• Main Authors: Meregalli, Mirella, Farini, Andrea, Belicchi, Marzia, Parolini, Daniele, Cassinelli, Letizia, Razini, Paola, Sitzia, Clementina, Torrente, Yvan
• Format: Article
• Language: English
• Subjects: Animals; Cell- and Tissue-Based Therapy; Cellular biology; Duchenne muscular dystrophy; Humans; Muscular dystrophy; Muscular Dystrophy, Duchenne - metabolism; Muscular Dystrophy, Duchenne - therapy; Mutation; Proteins; Stem Cell Transplantation; Stem cells; Stem Cells - cytology; Stem Cells - metabolism; therapy
• ISSN: 1742-464X; 1742-4658
• DOI: 10.1111/febs.12083

Muscular dystrophies are heritable and heterogeneous neuromuscular disorders characterized by the primary wasting of skeletal muscle, usually caused by mutations in the proteins forming the link between the cytoskeleton and the basal lamina. As a result of mutations in the dystrophin gene, Duchenne muscular dystrophy patients suffer from progressive muscle atrophy and an exhaustion of muscular regenerative capacity. No efficient therapies are available. The evidence that adult stem cells were capable of participating in the regeneration of more than their resident organ led to the development of potential stem cell treatments for degenerative disorder. In the present review, we describe the different types of myogenic stem cells and their possible use for the progression of cell therapy in Duchenne muscular dystrophy. Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy and unfortunately no effective therapy is available at present. As stem cells received much attention for their potential use in cell‐based therapies for human diseases, herein we described multiple types of resident and circulating myogenic stem cells, their characterization and their possible use to treat muscular dystrophies.