Table of Contents
ISRN Transplantation
Volume 2013, Article ID 582689, 25 pages
Review Article

Cell Transplantation and “Stem Cell Therapy” in the Treatment of Myopathies: Many Promises in Mice, Few Realities in Humans

1Neurosciences Division-Human Genetics, CHUQ Research Center-CHUL, Quebec, QC, Canada
2Unité de Recherche en Génétique Humaine, Centre Hospitalier de l'Université Laval, 2705 boulevard Laurier, Québec, QC, Canada G1V 4G2

Received 14 July 2013; Accepted 21 August 2013

Academic Editors: S. V. Brodsky, W. Lim, and C. Marchese

Copyright © 2013 Daniel Skuk. This is an open access article distributed under the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.


Myopathies produce deficits in skeletal muscle function and, in some cases, progressive and irreversible loss of skeletal muscles. The transplantation of myogenic cells, that is, cells able to differentiate into myofibers, is an experimental strategy for the potential treatment of some of these diseases. The objectives pursued by the transplantation of these cells are essentially three: (a) the fusion with the patient’s myofibers to obtain the expression of therapeutic proteins into them, (b) the neoformation of new functional myofibers in skeletal muscles that were too degenerated by the progressive degeneration, and (c) the formation of a new pool of healthy donor-derived satellite cells. Although the repertoire of myogenic cells appears to have expanded in recent years, myoblasts are the only cells that have been demonstrated to engraft in humans. The present work aims to make a comprehensive review of the subject, from its beginnings to recent advances, including the preclinical experience in different animal models and recent clinical findings.