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A beginner's guide to gene editing
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Date
2017-12-28
Authors
Harrison, Patrick T.
Hart, Stephen
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Wiley
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Abstract
Genome editing enables precise changes to be made in the genome of living cells. The technique was originally developed in the 1980′s but largely limited to use in mice. The discovery that a targeted double stranded break (DSB) at a unique site in the genome, close to the site to be changed, could substantially increase the efficiency of editing raised the possibility of using the technique in a broader range of animal models and potentially human cells. But the challenge was to identify reagents that could create targeted breaks at a unique genomic location with minimal off-target effects. In 2005, the demonstration that programmable zinc finger nucleases (ZFNs) could perform this task, led to a number of proof-of-concept studies, but a limitation was the ease with which effective ZFNs could be produced. In 2009, the development of TAL-effector nucleases (TALENs) increased the specificity of gene editing and the ease of design and production. However, it wasn't until 2013 and the development of the CRISPR Cas9/guideRNA that gene editing became a research tool that any lab could use.
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Keywords
Cas9 , CRISPR , Cystic fibrosis , gRNA , TALEN , ZFN , Genome editing , Gene editing
Citation
Harrison, P. T. and Hart, S. 'A beginner's guide to gene editing', Experimental Physiology, 103(4), pp. 439-448. doi:10.1113/EP086047
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© 2017 The Authors. Experimental Physiology © 2017 The Physiological Society. Published by Wiley. This is the peer reviewed version of the following article: Harrison, P. T. and Hart, S. 'A beginner's guide to gene editing', Experimental Physiology, 103(4), pp. 439-448, doi:10.1113/EP086047, which has been published in final form at http://dx.doi.org/10.1113/EP086047. This article may be used for non-commercial purposes in accordance with Wiley Terms and Conditions for Self-Archiving.