Pluripotent Stem Cell-Based Therapeutics for Muscular Dystrophies

Trends Mol Med. 2019 Sep;25(9):803-816. doi: 10.1016/j.molmed.2019.07.004.

Abstract

Pluripotent stem cells (PSCs) represent an attractive cell source for treating muscular dystrophies (MDs) since they easily allow for the generation of large numbers of highly regenerative myogenic progenitors. Using reprogramming technology, patient-specific PSCs have been derived for several types of MDs, and genome editing has allowed correction of mutations, opening the opportunity for their therapeutic application in an autologous transplantation setting. However, there has been limited progress on preclinical studies that validate the therapeutic potential of these gene corrected PSC-derived myogenic progenitors. In this review, we highlight the major research advances, challenges, and future prospects towards the development of PSC-based therapeutics for MDs.

Keywords: cell therapy; gene correction; muscular dystrophy; myogenic progenitors; pluripotent stem cells.

Publication types

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

MeSH terms

  • Animals
  • Biomarkers
  • Cell Differentiation
  • Cell- and Tissue-Based Therapy / methods
  • Disease Models, Animal
  • Gene Editing
  • Genetic Therapy
  • Humans
  • Induced Pluripotent Stem Cells / cytology
  • Induced Pluripotent Stem Cells / metabolism
  • Muscular Dystrophies / etiology
  • Muscular Dystrophies / therapy*
  • Myoblasts, Cardiac / cytology
  • Myoblasts, Cardiac / metabolism
  • Pluripotent Stem Cells / cytology*
  • Pluripotent Stem Cells / metabolism*
  • Regeneration
  • Stem Cell Transplantation* / methods
  • Transgenes

Substances

  • Biomarkers