Stem cell-based therapies for Duchenne muscular dystrophy

Congshan Sun, Carlo Serra, Gabsang Lee, Kathryn R. Wagner

Research output: Contribution to journalReview article

Abstract

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.

Original languageEnglish (US)
Article number113086
JournalExperimental Neurology
Volume323
DOIs
StatePublished - Jan 2020

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Induced Pluripotent Stem Cells
Pluripotent Stem Cells
Duchenne Muscular Dystrophy
Cell- and Tissue-Based Therapy
Stem Cells
Stem Cell Research
Dystrophin
Inborn Genetic Diseases
Muscular Dystrophies
Autologous Transplantation
Myoblasts
Cell Transplantation
Muscle Weakness
X Chromosome
Muscular Diseases
Bone Marrow Cells
Transplantation
Mutation
Genes
Proteins

Keywords

  • Duchenne muscular dystrophy (DMD)
  • hiPSC
  • Myogenic lineage specification
  • Stem cell therapy

ASJC Scopus subject areas

  • Neurology
  • Developmental Neuroscience

Cite this

Stem cell-based therapies for Duchenne muscular dystrophy. / Sun, Congshan; Serra, Carlo; Lee, Gabsang; Wagner, Kathryn R.

In: Experimental Neurology, Vol. 323, 113086, 01.2020.

Research output: Contribution to journalReview article

Sun, Congshan ; Serra, Carlo ; Lee, Gabsang ; Wagner, Kathryn R. / Stem cell-based therapies for Duchenne muscular dystrophy. In: Experimental Neurology. 2020 ; Vol. 323.
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