%0 Journal Article
%T Translational Research in Stem Cell Treatment of Neuromuscular Diseases
%A Hakan Orbay
%A Hiroshi Mizuno
%J ISRN Stem Cells
%D 2013
%R 10.1155/2013/947329
%X Neuromuscular diseases are a heterogeneous group of diseases that lead to significant disability in effected individuals. Pharmacological treatments failed to provide any significant improvement to date. Recently, the introduction of stem cells into the field of health sciences raised the hopes for a new treatment for neuromuscular diseases. In theory, stem cells, owing to their multilineage differentiation capacity, could differentiate into myofibers and neurons and replace the degenerated cells leading to recovery of the patients. Results obtained from the preclinical studies supported this theory. However, clinical trials with stem cells could not meet the expectations mainly because of early mortality, limited migration, and differentiation of the implanted cells. Modification of the stem cells before implantation, such as introduction of deficient genes or commitment to a precursor cell line provided little improvement. The biggest barrier to overcome for a successful of stem cell treatment, which also should be the focus of the future studies, is to increase the functional integration of the donor cells with the recipient tissues. Understanding the underlying pathogenic mechanisms of the neuromuscular diseases is essential to achieve this goal. 1. Introduction The term neuromuscular diseases define a wide range of conditions characterized by the weakness or wasting of the body muscles. Problems may primarily originate from the spinal cord, the peripheral nerves (neuropathies), the muscle fibers (myopathies), or the neuromuscular junction. Some of these diseases are hereditary, while others are acquired. The diagnosis is mainly done by clinical observation, electromyography, muscle biopsy, and in some instances molecular genetic studies. Some of the major types of neuromuscular diseases are amyotrophic lateral sclerosis (ALS), myasthenia gravis (MG), multiple sclerosis (MS), and muscular dystrophies (Duchenne¡¯s muscular dystrophy (DMD) and Becker¡¯s muscular dystrophy (BMD)). Despite the long lasting research on the pathogenesis and the molecular mechanisms of neuromuscular diseases, no satisfactory treatment has been offered yet for these diseases [1]. Stem cell research is relatively new in the medical field but holds a great potential for the treatment of a variety of diseases that remained untreatable so far. Stem cells are believed to exist in all tissues in human body and may be totipotent, pluripotent, or multipotent, depending on tissue type. Neuromuscular degeneration itself seems to promote proliferation, migration, and
%U http://www.hindawi.com/journals/isrn.stem.cells/2013/947329/