Myogenic Differentiation of ES Cells for Therapies in Neuromuscular Diseases: Progress to Date

Abstract

The neuromuscular disorders are a heterogeneous group of genetic diseases characterized by progressive degeneration and impaired regeneration of skeletal muscle, resulting in weakness. The mobility of patients is very reduced, leading, depending on the disease severity, to wheelchair dependency and reduced life expectancy and quality. Currently there are no proven treatments for these diseases, except for palliative measures to improve a patient’s quality of life. Nevertheless, cell therapy using embryonic or somatic stem cells is considered to offer the best potential for success, and many projects are now being undertaken to evaluate the therapeutic possibilities of this approach. Theoretically, due to their pluripotency, embryonic stem cells can give rise to any type of tissue, and raises the possibility of successfully treating many diseases. However, a simple injection of ES cells into various body locations in model organisms often leads to formation of undesirable teratomas and not to healthy new tissues. Accordingly, ES cells must be partially differentiated and selected prior to injection to increase the likelihood of implantation and growth of tissue exhibiting differentiation of the desired type. Although some modest advances have been achieved, to date the use of ES cells for therapy of neuromuscular disorders still remains a distant goal. In this review we mainly consider the role of embryonic stem cells in neuromuscular therapeutic approaches.