Genome engineering at the Dawn of the golden age

David Segal, Joshua F. Meckler

Research output: Contribution to journalArticle

81 Scopus citations

Abstract

Genome engineering-the ability to precisely alter the DNA information in living cells-is beginning to transform human genetics and genomics. Advances in tools and methods have enabled genetic modifications ranging from the "scarless" correction of a single base pair to the deletion of entire chromosomes. Targetable nucleases are leading the advances in this field, providing the tools to modify any gene in seemingly any organism with high efficiency. Targeted gene alterations have now been reported in more than 30 diverse species, ending the reign of mice as the exclusive model of mammalian genetics, and targetable nucleases have been used to modify more than 150 human genes and loci. A nuclease has also already entered clinical trials, signaling the beginning of genome engineering as therapy. The recent dramatic increase in the number of investigators using these techniques signifies a transition away from methods development toward a new age of exciting applications.

Original languageEnglish (US)
Pages (from-to)135-158
Number of pages24
JournalAnnual Review of Genomics and Human Genetics
Volume14
DOIs
StatePublished - Aug 2013

Keywords

  • Animal models
  • Gene targeting
  • Meganuclease
  • TALEN
  • Targeted mutagenesis
  • Transcription activator-like effector nuclease
  • ZFN
  • Zinc-finger nuclease

ASJC Scopus subject areas

  • Genetics(clinical)
  • Genetics
  • Molecular Biology

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