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Gene therapy intervention in neovascular eye disease: a recent update


Lin, F-L and Wang, P-Y and Chuang, Y-F and Wang, J-H and Wong, VHY and Bui, BV and Liu, G-S, Gene therapy intervention in neovascular eye disease: a recent update, Molecular Therapy, 28, (10) pp. 1-19. ISSN 1525-0016 (2020) [Refereed Article]

Copyright Statement

Copyright 2020 The American Society of Gene and Cell Therapy

DOI: doi:10.1016/j.ymthe.2020.06.029


Aberrant growth of blood vessels (neovascularization) is a key feature of severe eye diseases that can cause legal blindness, including neovascular age-related macular degeneration (nAMD) and diabetic retinopathy (DR). The development of anti-vascular endothelial growth factor (VEGF) agents has revolutionized the treatment of ocular neovascularization. Novel proangiogenic targets, such as angiopoietin and platelet-derived growth factor (PDGF), are under development for patients who respond poorly to anti-VEGF therapy and to reduce adverse effects from long-term VEGF inhibition. A rapidly advancing area is gene therapy, which may provide significant therapeutic benefits. Viral vector-mediated transgene delivery provides the potential for continuous production of antiangiogenic proteins, which would avoid the need for repeated anti-VEGF injections. Gene silencing with RNA interference to target ocular angiogenesis has been investigated in clinical trials. Proof-of-concept gene therapy studies using gene-editing tools such as CRISPR-Cas have already been shown to be effective in suppressing neovascularization in animal models, highlighting the therapeutic potential of the system for treatment of aberrant ocular angiogenesis. This review provides updates on the development of anti-VEGF agents and novel antiangiogenic targets. We also summarize current gene therapy strategies already in clinical trials and those with the latest approaches utilizing CRISPR-Cas gene editing against aberrant ocular neovascularization.

Item Details

Item Type:Refereed Article
Keywords:ocular neovascularisation, VEGF, age-related macular degeneration, diabetic retinopathy, eye, gene therapy, neovascularization
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:Lin, F-L (Dr Fan-Li Lin)
UTAS Author:Chuang, Y-F (Dr Yu-Fan Chuang)
UTAS Author:Liu, G-S (Associate Professor Guei-Sheung Liu)
ID Code:139997
Year Published:2020
Funding Support:National Health and Medical Research Council (1185600)
Web of Science® Times Cited:17
Deposited By:Menzies Institute for Medical Research
Deposited On:2020-07-22
Last Modified:2022-08-26

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