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Gene therapy for diabetic retinopathy: are we ready to make the leap from bench to bedside?

Wang, J-H, Ling, D, Tu, L, van Wijngaarden, P, Dusting, GJ and Liu, G-S ORCID: 0000-0003-3379-724X 2017 , 'Gene therapy for diabetic retinopathy: are we ready to make the leap from bench to bedside?' , Pharmacology and Therapeutics, vol. 173 , pp. 1-18 , doi:

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Diabetic retinopathy (DR), a chronic and progressive complication of diabetes mellitus, is a sight-threatening disease characterized in the early stages by neuronal and vascular dysfunction in the retina, and later by neovascularization that further damages vision. A major contributor to the pathology is excess production of vascular endothelial growth factor (VEGF), a growth factor that induces formation of new blood vessels and increases permeability of existing vessels. Despite the recent availability of effective treatments for the disease, including laser photocoagulation and therapeutic VEGF antibodies, DR remains a significant cause of vision loss worldwide. Existing anti-VEGF agents, though generally effective, are limited by their short therapeutic half-lives, necessitating frequent intravitreal injections and the risk of attendant adverse events. Management of DR with gene therapies has been proposed for several years, and pre-clinical studies have yielded enticing findings. Gene therapy holds several advantages over conventional treatments for DR, such as a longer duration of therapeutic effect, simpler administration, the ability to intervene at an earlier stage of the disease, and potentially fewer side-effects. In this review, we summarize the current understanding of the pathophysiology of DR and provide an overview of research into DR gene therapies. We also examine current barriers to the clinical application of gene therapy for DR and evaluate future prospects for this approach.

Item Type: Article
Authors/Creators:Wang, J-H and Ling, D and Tu, L and van Wijngaarden, P and Dusting, GJ and Liu, G-S
Keywords: diabetic retinopathy, gene therapies, diabetic macular edema, neuronal degeneration, vascular dysfunction, vascular endothelial growth factor
Journal or Publication Title: Pharmacology and Therapeutics
Publisher: Pergamon-Elsevier Science Ltd
ISSN: 0163-7258
DOI / ID Number:
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Copyright 2017 Elsevier Inc.

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