Challenges in cell transplantation for muscular dystrophy

Exp Cell Res. 2021 Dec 1;409(1):112908. doi: 10.1016/j.yexcr.2021.112908. Epub 2021 Nov 1.


For decades now, cell transplantation has been considered a possible therapeutic strategy for muscular dystrophy, but failures have largely outnumbered success or at least encouraging outcomes. In this review we will briefly recall the history of cell transplantation, discuss the peculiar features of skeletal muscle, and dystrophic skeletal muscle in particular, that make the procedure complicated and inefficient. As there are many recent and exhaustive reviews on the various myogenic cell types that have been or will be transplanted, we will only briefly describe them and refer the reader to these reviews. Finally, we will discuss possible strategies to overcome the hurdles that prevent biological efficacy and hence clinical success.

Keywords: Cell transplantation; Clinical trials; Muscular dystrophies; Myogenic progenitor cells.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Cell Differentiation / physiology
  • Cell Transplantation / methods*
  • Humans
  • Muscle Development / physiology
  • Muscle, Skeletal / cytology*
  • Muscle, Skeletal / pathology*
  • Muscular Dystrophy, Duchenne / therapy*