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Inherited retinal diseases: Therapeutics, clinical trials and end points—A review

Georgiou, M; Fujinami, K; Michaelides, M; (2021) Inherited retinal diseases: Therapeutics, clinical trials and end points—A review. Clinical & Experimental Ophthalmology 10.1111/ceo.13917. (In press). Green open access

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Abstract

Inherited retinal diseases (IRDs) are a clinically and genetically heterogeneous group of disorders characterised by photoreceptor degeneration or dysfunction. These disorders typically present with severe vision loss that can be progressive, with disease onset ranging from congenital to late adulthood. The advances in genetics, retinal imaging and molecular biology, have conspired to create the ideal environment for establishing treatments for IRDs, with the first approved gene therapy and the commencement of multiple clinical trials. The scope of this review is to familiarise clinicians and scientists with the current management and the prospects for novel therapies for: (1) macular dystrophies, (2) cone and cone‐rod dystrophies, (3) cone dysfunction syndromes, (4) Leber congenital amaurosis, (5) rod‐cone dystrophies, (6) rod dysfunction syndromes and (7) chorioretinal dystrophies. We also briefly summarise the investigated end points for the ongoing trials.

Type: Article
Title: Inherited retinal diseases: Therapeutics, clinical trials and end points—A review
Location: Australia
Open access status: An open access version is available from UCL Discovery
DOI: 10.1111/ceo.13917
Publisher version: https://doi.org/10.1111/ceo.13917
Language: English
Additional information: Copyright © 2021 The Authors. Clinical & Experimental Ophthalmology published by John Wiley & Sons Australia, Ltd on behalf of Royal Australian and New Zealand College of Ophthalmologists. This is an open access article (http://creativecommons.org/licenses/by/4.0/) under the terms of the Creative Commons Attribution License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited.
Keywords: gene therapy, inherited retinal disease, pharmacological therapy, retina, stem cell
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 Brain Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > Institute of Ophthalmology
URI: https://discovery.ucl.ac.uk/id/eprint/10124889
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