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Updates on gene therapy for diabetic retinopathy


Wang, J-H and Roberts, GE and Liu, G-S, Updates on gene therapy for diabetic retinopathy, Current Diabetes Reports, 20, (7) Article 22. ISSN 1534-4827 (2020) [Refereed Article]

Copyright Statement

Copyright Springer Science+Business Media, LLC, part of Springer Nature 2020

DOI: doi:10.1007/s11892-020-01308-w


Purpose of review: Diabetic retinopathy (DR), a leading cause of visual impairment in the developed country, is characterized by vascular lesions and neuronal damage of the retina. Treatment options for this condition are currently limited. The advent of therapy targeting vascular endothelial growth factor (VEGF) demonstrated significant benefits to patients with DR. However, this treatment is limited by its short half-life and requirement for frequent invasive intravitreal injections. In addition, many patients failed to achieve clinically significant improvement in visual function. Gene therapy has the potential to provide an alternative treatment for DR with distinct advantages, such as longer therapeutic effect, less injection frequency, ability to intervene at disease onset, and potentially fewer side effects.

Recent findings: Strategies for gene therapy in DR, stemming from the current understanding of the disease pathogenesis, focus on the inhibition of neovascularization and protection of neurovascular degeneration in the retina. Studies with promising results have mainly focussed on animal models due to efficacy and safety concerns, despite a number of successful preclinical studies using adeno-associated virus-mediated transduction to treat both vascular dysfunction and neuronal degeneration. With the optimization of delivery vectors, transgene regulation, and outcome measure, gene therapy will potentially become available for patients with DR.

Summary: This review provides an update on the current strategies utilized in DR gene therapy research. Several barriers to the clinical application of gene therapy for DR are highlighted, and future directions for this research are proposed.

Item Details

Item Type:Refereed Article
Keywords:angiogenesis, diabetes, gene therapy, retinopathy, diabetic retinopathy
Research Division:Biomedical and Clinical Sciences
Research Group:Medical biotechnology
Research Field:Gene and molecular therapy
Objective Division:Health
Objective Group:Clinical health
Objective Field:Clinical health not elsewhere classified
UTAS Author:Liu, G-S (Associate Professor Guei-Sheung Liu)
ID Code:139226
Year Published:2020
Funding Support:National Health and Medical Research Council (1185600)
Web of Science® Times Cited:9
Deposited By:Menzies Institute for Medical Research
Deposited On:2020-06-02
Last Modified:2022-08-26

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