The efficacy of naïve versus modified mesenchymal stem cells in improving muscle function in duchenne muscular dystrophy: A systematic review

Oscar Yuan Jie Shen, Yi Fan Chen, Hong Tao Xu, Chien Wei Lee

Research output: Contribution to journalReview articlepeer-review

Abstract

As one of the most common genetic conditions, Duchenne muscular dystrophy (DMD) is a fatal disease caused by a recessive mutation resulting in muscle weakness in both voluntary and involuntary muscles and, eventually, in death because of cardiovascular failure. Currently, there is no pharmacologically curative treatment of DMD, but there is evidence supporting that mesenchy-mal stem cells (MSCs) are a novel solution for treating DMD. This systematic review focused on elucidating the therapeutic efficacy of MSCs on the DMD in vivo model. A key issue of previous studies was the material‐choice, naïve MSCs or modified MSCs; modified MSCs are activated by culture methods or genetic modification. In summary, MSCs seem to improve pulmonary and cardiac functions and thereby improve survival regardless of them being naïve or modified. The improved function of distal skeletal muscles was observed only with primed MSCs treatment but not naïve MSCs. While MSCs can provide significant benefits to DMD mouse models, there is little to no data on the results in human patients. Due to the limited number of human studies, the differences in study design, and the insufficient understanding of mechanisms of action, more rigorous comparative trials are needed to elucidate which types of MSCs and modifications have optimal therapeutic potential.

Original languageEnglish
Article number1097
JournalBiomedicines
Volume9
Issue number9
DOIs
Publication statusPublished - Sep 2021

Keywords

  • Contraction‐induced injury
  • Fibrosis
  • Mesenchymal stem cells Duchenne muscular dystrophy
  • Muscle function
  • Regenerative cell therapy

ASJC Scopus subject areas

  • Medicine (miscellaneous)
  • Biochemistry, Genetics and Molecular Biology(all)

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