Genome editing: the breakthrough technology for inherited retinal disease?

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Title: Genome editing: the breakthrough technology for inherited retinal disease?
Authors: Smith, Andrew J.
Carter, Stephen P.
Kennedy, Breandán
Permanent link: http://hdl.handle.net/10197/10555
Date: 11-Jul-2017
Online since: 2019-05-21T07:47:15Z
Abstract: Introduction: Genetic alterations resulting in a dysfunctional retinal pigment epithelium and/or degenerating photoreceptors cause impaired vision. These juxtaposed cells in the retina of the posterior eye are crucial for the visual cycle or phototransduction. Deficits in these biochemical processes perturb neural processing of images capturing the external environment. Notably, there is a distinct lack of clinically approved pharmacological, cell- or gene-based therapies for inherited retinal disease. Gene editing technologies are rapidly advancing as a realistic therapeutic option. Areas Covered: Recent discovery of endonuclease-mediated gene editing technologies has culminated in a surge of investigations into their therapeutic potential. In this review, the authors discuss gene editing technologies and their applicability in treating inherited retinal diseases, the limitations of the technology and the research obstacles to overcome before editing a patient’s genome becomes a viable treatment option. Expert Opinion: The ability to strategically edit a patient’s genome constitutes a treatment revolution. However, concerns remain over the safety and efficacy of either transplanting iPSC-derived retinal cells following ex vivo gene editing, or with direct gene editing in vivo. Ultimately, further refinements to improve efficacy and safety profiles are paramount for gene editing to emerge as a widely available treatment option.
Funding Details: Health Research Board
Irish Research Council
Wellcome Trust
Type of material: Journal Article
Publisher: Taylor & Francis
Journal: Expert Opinion on Biological Therapy
Volume: 17
Issue: 10
Start page: 1245
End page: 1254
Copyright (published version): 2017 Taylor & Francis
Keywords: RetinaPhotoreceptorRetinal pigment epitheliumInherited retinal diseaseGene editingCRISPR
DOI: 10.1080/14712598.2017.1347629
Language: en
Status of Item: Peer reviewed
Appears in Collections:Biomolecular and Biomedical Science Research Collection

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