Stem cells for the treatment of Muscular dystrophy: therapeutic perspectives

Muscular dystrophies are caused by progressive degeneration of skeletal muscle fibers. Lack of one of several proteins, increases the probability of damage during contraction, and eventually leads to fiber degeneration. Current therapeutic approaches involve steroids, and result in modest beneficial effects. Novel experimental approaches can be schematically grouped in three major areas: gene therapy, pharmacological approaches, and cell therapy. The topic of this chapter is the recent identification of novel types of stem cells which open new perspectives for cell therapy. It deals with the use of stem cells to treat muscular dystrophy and reviews previous cell therapy trials in mice and patients through the use of donor myoblasts, explaining the likely reasons for their failure, and briefly outlining the results on satellite cells. This chapter explores the identification of myogenic progenitors in the bone marrow and the related trials in animal models to treat dystrophy by bone marrow transplantation, and then describes recently identified stem/progenitor cells in relationship to their ability to home within a dystrophic muscle, and to differentiate into skeletal muscle cells. Different known features of various stem cells are compared in this perspective, and the few available examples of their use in animal models of dystrophy are reported. On the basis of current knowledge and due to the rapid advance in stem cell biology, a prediction of clinical translation for these cell therapy protocols is outlined. © 2009 Copyright © 2009 Elsevier Inc. All rights reserved.

ISBN:978-0-12-374729-7

Jaar van publicatie:2009