Research and Therapeutic Approaches in Stem Cell Genome Editing by CRISPR Toolkit

Molecules. 2023 Feb 20;28(4):1982. doi: 10.3390/molecules28041982.

Abstract

The most widely used genome editing toolkit is CRISPR (clustered regularly interspaced short palindromic repeats). It provides the possibility of replacing and modifying DNA and RNA nucleotides. Furthermore, with advancements in biological technology, inhibition and activation of the transcription of specific gene(s) has become possible. Bioinformatics tools that target the evolution of CRISPR-associated protein 9 (Cas9) turn this protein into a vehicle that is specific for a DNA or RNA region with single guide RNA (sgRNA). This toolkit could be used by researchers to investigate the function of stem cell gene(s). Here, in this review article, we cover recent developments and applications of this technique in stem cells for research and clinical purposes and discuss different CRISPR/Cas technologies for knock-out, knock-in, activation, or inhibition of gene expression. Additionally, a comparison of several deliveries and off-target detecting strategies is discussed.

Keywords: CRISPR; bioinformatics; cell therapy; genome editing; stem cells.

Publication types

  • Review

MeSH terms

  • CRISPR-Cas Systems*
  • DNA / genetics
  • Gene Editing* / methods
  • RNA
  • Stem Cells

Substances

  • RNA
  • DNA

Grants and funding

The research did not receive any specific grant from funding agencies in the public, commercial, or not-for-profit sectors.