Genome editing: the breakthrough technology for inherited retinal disease?

DC FieldValueLanguage
dc.contributor.authorSmith, Andrew J.-
dc.contributor.authorCarter, Stephen P.-
dc.contributor.authorKennedy, Breandán-
dc.date.accessioned2019-05-21T07:47:15Z-
dc.date.available2019-05-21T07:47:15Z-
dc.date.copyright2017 Taylor & Francisen_US
dc.date.issued2017-07-11-
dc.identifier.citationExpert Opinion on Biological Therapyen_US
dc.identifier.issn1471-2598-
dc.identifier.urihttp://hdl.handle.net/10197/10555-
dc.description.abstractIntroduction: 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.en_US
dc.description.sponsorshipHealth Research Boarden_US
dc.description.sponsorshipIrish Research Councilen_US
dc.description.sponsorshipWellcome Trusten_US
dc.format.mediumPrint-Electronic-
dc.language.isoenen_US
dc.publisherTaylor & Francisen_US
dc.rightsThis is an Accepted Manuscript of an article published by Taylor & Francis in Expert Opinion on Biological Therapy on 11 July 2017, available online: http://www.tandfonline.com/10.1080/14712598.2017.1347629.en_US
dc.subjectRetinaen_US
dc.subjectPhotoreceptoren_US
dc.subjectRetinal pigment epitheliumen_US
dc.subjectInherited retinal diseaseen_US
dc.subjectGene editingen_US
dc.subjectCRISPRen_US
dc.titleGenome editing: the breakthrough technology for inherited retinal disease?en_US
dc.typeJournal Articleen_US
dc.internal.authorcontactotherbrendan.kennedy@ucd.ieen_US
dc.statusPeer revieweden_US
dc.identifier.volume17en_US
dc.identifier.issue10en_US
dc.identifier.startpage1245en_US
dc.identifier.endpage1254en_US
dc.identifier.doi10.1080/14712598.2017.1347629-
dc.neeo.contributorSmith|Andrew J.|aut|-
dc.neeo.contributorCarter|Stephen P.|aut|-
dc.neeo.contributorKennedy|Breandán|aut|-
dc.description.othersponsorshipFighting Blindnessen_US
dc.date.updated2019-05-17T16:34:50Z-
dc.identifier.grantidWT106820MA-
dc.identifier.grantidGOIPG/2014/683-
dc.identifier.grantidMRCG/2014/3-
item.fulltextWith Fulltext-
item.grantfulltextopen-
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