To CRISPR and beyond: the evolution of genome editing in stem cells

Regen Med. 2016 Dec;11(8):801-816. doi: 10.2217/rme-2016-0107. Epub 2016 Dec 1.


The goal of editing the genomes of stem cells to generate model organisms and cell lines for genetic and biological studies has been pursued for decades. There is also exciting potential for future clinical impact in humans. While recent, rapid advances in targeted nuclease technologies have led to unprecedented accessibility and ease of gene editing, biology has benefited from past directed gene modification via homologous recombination, gene traps and other transgenic methodologies. Here we review the history of genome editing in stem cells (including via zinc finger nucleases, transcription activator-like effector nucleases and CRISPR-Cas9), discuss recent developments leading to the implementation of stem cell gene therapies in clinical trials and consider the prospects for future advances in this rapidly evolving field.

Keywords: CRISPR-Cas9; TALEN; ZFN; gene therapy; genome editing; stem cells.

Publication types

  • Review
  • Research Support, N.I.H., Extramural

MeSH terms

  • Animals
  • Clustered Regularly Interspaced Short Palindromic Repeats*
  • Endonucleases / genetics*
  • Gene Editing*
  • Genetic Engineering / methods*
  • Genome, Human*
  • Humans
  • Stem Cells / cytology
  • Stem Cells / metabolism*


  • Endonucleases