Advancing Mesenchymal Stem Cell Therapy with CRISPR/Cas9 for Clinical Trial Studies

Adv Exp Med Biol. 2020:1247:89-100. doi: 10.1007/5584_2019_459.

Abstract

Currently, regenerative medicine and cellular-based therapy have been in the center of attention worldwide in advanced medical technology. Mesenchymal stem cell (MSC) as a suitable stem cell source for cell-based therapy has been shown to be safe and effective in multiple clinical trial studies (CTSs) of several diseases. Despite the advantages, MSC needs more investigation to enhance its therapeutic application. The CRISPR/Cas system is a novel technique for editing of genes that is being explored as a means to improve MSCs therapeutic usage. In this study, we review the recent studies that explore CRISPR potency in gene engineering of MSCs, which have great relevance in MSC-based therapies. However, CRISPR/Cas technology make possible specific targeting of loci in target genes, but next-generation MSC-based therapies to achieve extensive clinical application need dedicated efforts.

Keywords: CRISPR; Cell therapy; Gene editing; Mesenchymal stem cell.

Publication types

  • Review

MeSH terms

  • CRISPR-Cas Systems*
  • Clinical Trials as Topic*
  • Gene Editing
  • Humans
  • Mesenchymal Stem Cell Transplantation / methods*
  • Mesenchymal Stem Cells / metabolism*
  • Regenerative Medicine