Perspectives of stem cell therapy in Duchenne muscular dystrophy.

Abstract

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.

• Authors: Mirella Meregalli, Andrea Farini, Marzia Belicchi, Daniele Parolini, Letizia Cassinelli, Paola Razini, Clementina Sitzia, Yvan Torrente
• Journal: The FEBS journal (FEBS J) Vol. 280 Issue 17 Pg. 4251-62 (Sep 2013) ISSN: 1742-4658 [Electronic] England
• PMID: 23206279 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't, Review)
• Copyright: © 2012 The Authors Journal compilation © 2012 FEBS.

Topics

• Animals
• Cell- and Tissue-Based Therapy
• Humans
• Muscular Dystrophy, Duchenne (metabolism, therapy)
• Stem Cell Transplantation
• Stem Cells (cytology, metabolism)