Pluripotent Stem Cell-Based Therapeutics for Muscular Dystrophies

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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.

Original languageEnglish (US)
Pages (from-to)803-816
Number of pages14
JournalTrends in Molecular Medicine
Volume25
Issue number9
DOIs
StatePublished - Sep 2019

Bibliographical note

Funding Information:
R.C.R.P. is supported by National Institutes of Health (NIH) grants R01 AR055299 and R01 AR071439 , ADVault, Inc. and MyDirectives.com . M.K. is supported by NIH R01 AR055685 . We are thankful to Cynthia Faraday for graphical design.

Publisher Copyright:
© 2019 Elsevier Ltd

Keywords

  • cell therapy
  • gene correction
  • muscular dystrophy
  • myogenic progenitors
  • pluripotent stem cells

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