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

Kuang Yui Chen, Paul S Knoepfler

Research output: Contribution to journalReview article

6 Scopus citations

Abstract

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.

Original languageEnglish (US)
Pages (from-to)801-816
Number of pages16
JournalRegenerative Medicine
Volume11
Issue number8
DOIs
StatePublished - Dec 1 2016

Keywords

  • CRISPR-Cas9
  • gene therapy
  • genome editing
  • stem cells
  • TALEN
  • ZFN

ASJC Scopus subject areas

  • Embryology
  • Biomedical Engineering

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