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Gene therapy intervention in neovascular eye disease: a recent update
Citation
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
Abstract
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: | 2 |
| Deposited By: | Menzies Institute for Medical Research |
| Deposited On: | 2020-07-22 |
| Last Modified: | 2020-08-07 |
| Downloads: | 0 |
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