Stem cell-based therapies for Duchenne muscular dystrophy

Exp Neurol. 2020 Jan:323:113086. doi: 10.1016/j.expneurol.2019.113086. Epub 2019 Oct 19.


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, we discuss the existing cell therapy research, especially stem cell-based, which utilize myoblasts, satellite cells, bone marrow cells, mesoangioblasts and CD133+ cells. Finally, we focus on 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. 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, we summarize the current methods of hPSCs myogenic commitment/differentiation, and describe the current status of hPSC-derived myogenic cell transplantation.

Keywords: Duchenne muscular dystrophy (DMD); Myogenic lineage specification; Stem cell therapy; hiPSC.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Cell Differentiation / physiology
  • Humans
  • Muscular Dystrophy, Duchenne / therapy*
  • Myoblasts / cytology*
  • Myoblasts / transplantation
  • Pluripotent Stem Cells / cytology*
  • Stem Cell Transplantation / methods*