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

Mo Li, Keiichiro Suzuki, Na Young Kim, Guang Hui Liu, Juan Carlos Izpisua Belmonte*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

93 Scopus citations

Abstract

Human pluripotent stem cells (hPSCs) offer unprecedented opportunities to study cellular differentiation and modelhuman 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.Wediscuss the features and limitations of these technologies, as well as how these factors influence the utility of these tools in basic research and therapies.

Original languageEnglish (US)
Pages (from-to)4594-4599
Number of pages6
JournalJournal of Biological Chemistry
Volume289
Issue number8
DOIs
StatePublished - Feb 21 2014
Externally publishedYes

ASJC Scopus subject areas

  • Biochemistry
  • Molecular Biology
  • Cell Biology

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