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Trends Biotechnol. 2018 Aug;36(8):770-786. doi: 10.1016/j.tibtech.2018.03.004. Epub 2018 Apr 21.

Strategies for In Vivo Genome Editing in Nondividing Cells.

Author information

1
Department of Development and Regeneration, KU Leuven-Stamcelinstituut, Leuven, Belgium.
2
Department of Stem Cells and Developmental Biology, Cell Science Research Center, Royan Institute for Stem Cell Biology and Technology, ACECR, Tehran, Iran.
3
Department of Stem Cells and Developmental Biology, Cell Science Research Center, Royan Institute for Stem Cell Biology and Technology, ACECR, Tehran, Iran; Department of Developmental Biology, University of Science and Culture, Tehran, Iran. Electronic address: baharvand@royaninstitute.org.
4
Department of Development and Regeneration, KU Leuven-Stamcelinstituut, Leuven, Belgium. Electronic address: catherine.verfaillie@kuleuven.be.

Abstract

Programmable nucleases, including zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated 9 (Cas9), have enhanced our ability to edit genomes by the sequence-specific generation of double-strand breaks (DSBs) with subsequent homology-directed repair (HDR) of the DSB. However, the efficiency of the HDR pathway is limited in nondividing cells, which encompass most of the cells in the body. Therefore, the HDR-mediated genome-editing approach has limited in vivo applicability. Here, we discuss a mutation type-oriented viewpoint of strategies devised over the past few years to circumvent this problem, along with their possible applications and limitations.

KEYWORDS:

gene editing; homology directed repair; in vivo; non-dividing cells; non-homologous end joining

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