Scientists of John Hopkins University School of Medicine summarize the current methods of human pluripotent stem cells (HPSCs) myogenic commitment/differentiation, and describe the current status of hPSC-derived myogenic cell transplantation.
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. DMD is an X chromosome linked disease that occurs to 1 in 3500 to 1 in 5000 boys.
The cause of DMD is a mutation in the dystrophin gene, whose encoded protein provides both structural support and cell signaling capabilities. So far, there are very limited therapeutic options available and there is no cure for this disease.
In this review, is discussed the existing cell therapy research, especially stem cell-based, which utilize myoblasts, satellite cells, bone marrow cells, mesoangioblasts and CD133+ cells.
Finally, human pluripotent stem cells (hPSCs) are focused, which hold great potential in treating DMD. hPSCs can be used for autologous transplantation after being specified to a myogenic lineage.
Over the last few years, there has been a rapid development of isolation, as well as differentiation, techniques in order to achieve effective transplantation results of myogenic cells specified from hPSCs.
In this review, the current methods of hPSCs myogenic commitment/differentiation are summarized and described the current status of hPSC-derived myogenic cell transplantation.