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A beginner's guide to gene editing

Harrison, PT; Hart, S; (2018) A beginner's guide to gene editing. Experimental Physiology , 103 (4) pp. 439-448. 10.1113/EP086047. Green open access

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Abstract

Genome editing enables precise changes to be made in the genome of living cells. The techniquewas originally developed in the 1980s but largely limited to use in mice. The discovery that atargeted double-stranded break 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 techniquein a broader range of animal models and, potentially, human cells. But the challenge was toidentify reagents that could create targeted breaks at a unique genomic location with minimaloff-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 easewith 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 was not until 2013 and the development of the clustered regularly interspaced shortpalindromic repeat (CRISPR) Cas9/guide RNA that gene editing became a research tool that anylaboratory could use.

Type: Article
Title: A beginner's guide to gene editing
Open access status: An open access version is available from UCL Discovery
DOI: 10.1113/EP086047
Publisher version: http://doi.org/10.1113/EP086047
Language: English
Additional information: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
Keywords: Cas9, CRISPR, cystic fibrosis, gene editing, guide RNA, TAL-effector nuclease, zinc finger nuclease
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Genetics and Genomic Medicine Dept
URI: https://discovery.ucl.ac.uk/id/eprint/10048265
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