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Genome engineering in ophthalmology: Application of CRISPR/Cas to the treatment of eye disease


Hung, SSC and McCaughey, T and Swann, OG and Pebay, A and Hewitt, AW, Genome engineering in ophthalmology: Application of CRISPR/Cas to the treatment of eye disease, Progress in Retinal and Eye Research, 53 pp. 1-20. ISSN 1350-9462 (2016) [Refereed Article]

Copyright Statement

Copyright 2016 Elsevier Ltd.

DOI: doi:10.1016/j.preteyeres.2016.05.001


The Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR) and CRISPR-associated protein (Cas) system has enabled an accurate and efficient means to edit the human genome. Rapid advances in this technology could results in imminent clinical application, and with favourable anatomical and immunological profiles, ophthalmic disease will be at the forefront of such work. There have been a number of breakthroughs improving the specificity and efficacy of CRISPR/Cas-mediated genome editing. Similarly, better methods to identify off-target cleavage sites have also been developed. With the impending clinical utility of CRISPR/Cas technology, complex ethical issues related to the regulation and management of the precise applications of human gene editing must be considered. This review discusses the current progress and recent breakthroughs in CRISPR/Cas-based gene engineering, and outlines some of the technical issues that must be addressed before gene correction, be it in vivo or in vitro, is integrated into ophthalmic care. We outline a clinical pipeline for CRISPR-based treatments of inherited eye diseases and provide an overview of the important ethical implications of gene editing and how these may influence the future of this technology.

Item Details

Item Type:Refereed Article
Keywords:Cas9, Cpf1, Ethics, Gene editing, Genetic ophthalmology, Inherited retinal disease
Research Division:Biomedical and Clinical Sciences
Research Group:Ophthalmology and optometry
Research Field:Ophthalmology
Objective Division:Health
Objective Group:Clinical health
Objective Field:Clinical health not elsewhere classified
UTAS Author:Swann, OG (Miss Olivia Swann)
UTAS Author:Hewitt, AW (Professor Alex Hewitt)
ID Code:111188
Year Published:2016
Web of Science® Times Cited:29
Deposited By:Menzies Institute for Medical Research
Deposited On:2016-09-01
Last Modified:2022-08-25

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