CRISPR/Cas9 system: a powerful technology for in vivo and ex vivo gene therapy

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28 Scopus citations

Abstract

CRISPR/Cas9 is a versatile genome-editing tool which is widely used for modifying the genome of both prokaryotic and eukaryotic organisms for basic research and applications. An increasing number of reports have demonstrated that CRISPR/Cas9-mediated genome editing is a powerful technology for gene therapy. Here, we review the recent advances in CRISPR/Cas9-mediated gene therapy in animal models via different strategies and discuss the challenges as well as future prospects.

Original languageEnglish
Pages (from-to)468-475
Number of pages8
JournalScience China Life Sciences
Volume60
Issue number5
DOIs
StatePublished - 1 May 2017

Keywords

  • CRISPR/Cas9
  • ex vivo
  • gene therapy
  • in vivo

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