A cut above the rest: targeted genome editing technologies in human pluripotent stem cells

J Biol Chem. 2014 Feb 21;289(8):4594-9. doi: 10.1074/jbc.R113.488247. Epub 2013 Dec 20.


Human pluripotent stem cells (hPSCs) offer unprecedented opportunities to study cellular differentiation and model human diseases. The ability to precisely modify any genomic sequence holds the key to realizing the full potential of hPSCs. Thanks to the rapid development of novel genome editing technologies driven by the enormous interest in the hPSC field, genome editing in hPSCs has evolved from being a daunting task a few years ago to a routine procedure in most laboratories. Here, we provide an overview of the mainstream genome editing tools, including zinc finger nucleases, transcription activator-like effector nucleases, clustered regularly interspaced short palindromic repeat/CAS9 RNA-guided nucleases, and helper-dependent adenoviral vectors. We discuss the features and limitations of these technologies, as well as how these factors influence the utility of these tools in basic research and therapies.

Keywords: CRISPR/CAS9; Embryonic Stem Cell; Gene Therapy; Genome Editing; HDAdV; Induced Pluripotent Stem Cell (iPSC); Stem Cells; TALEN; ZFN.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Deoxyribonucleases / metabolism
  • Genetic Engineering / methods*
  • Genome, Human / genetics*
  • Humans
  • Pluripotent Stem Cells / metabolism*
  • RNA Editing / genetics*


  • Deoxyribonucleases