STEM CELLS BASED THERAPY IN DUCHENNE MUSCULAR DYSTROPHY
Muscular dystrophies (MDs) are a group of more than 50 heterogeneous genetic diseases, often presented in children marked by degeneration of skeletal muscle and progressive weakness. The different MDs vary in terms of groups of muscles involved, age at disease onset, progression, and ultimate level of disability. Furthermore, several MDs show compromised physiology of other organs, such as the heart and brain in Duchenne muscular dystrophy (DMD) The most common form of MD is DMD, a fatal disease affecting around 1 in 3,500 to 1 in 5,000 live male births, Boys with DMD usually lose the ability to walk in early teenage years, lose the ability to feed themselves in late teenage years , and die from respiratory insufficiency or cardiomyopathy in early adulthood, Current standard of care includes the use of corticosteroids, cardioprotective treatment, ventilatory support, and physical therapy, However, these treatments have limitations and side effects, and are only able to delay the progression of the disease
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Muscular dystrophies are a group of genetic muscle disorders that cause progressive muscle weakness and degeneration. Within this group, Duchenne muscular dystrophy (DMD) is the most common and one of the most severe. So far, there are very limited therapeutic options available and there is no cure for this disease. But there is a hope with cell therapy especially stem cell - based, which utilize myoblasts, satellite cells, bone marrow cells, mesoangioblasts and CD133+ cells. And finally the human pluripotent stem cells (hPSCs) which hold great potential in treating DMD. hPSCs can be used for autologous transplantation after being specified to a myogenic lineage. We are going to summarize the current methods of hPSCs myogenic commitment, differentiation, and describe the current status of hPSC-derived myogenic cell transplantation.
