Cited by 74Retinal Diseases and TreatmentsRetinal Development and DisordersAngiogenesis and VEGF in Cancer

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Gene Therapy Intervention in Neovascular Eye Disease: A Recent Update

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Lin, Fan-Li, Wang, Peng-Yuan, Chuang, Yu-Fan, Wang, Jiang-Hui, Wong, Vickie H.Y., Bui, Bang V., Liu, Guei-Sheung·2020·Molecular Therapy

DOI 10.1016/j.ymthe.2020.06.029PMC PMC7544979

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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.

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Polymer- and lipid-based nanocarriers for ocular drug delivery: Current status and future perspectives
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Authors: Lin, F.-L., Wang, P.-Y., Chuang, Y.-F., Wang, J.-H., Wong, V. H. Y., Bui, B. V., & Liu, G.-S.
Affiliations: University of Tasmania (AU)
Chinese Academy of Sciences (CN)
The Royal Victorian Eye & Ear Hospital (AU)
The University of Melbourne (AU)
Corresponding author: Wang, Peng-Yuan
Liu, Guei-Sheung
ORCID: Lin, Fan-Li
Wang, Peng-Yuan
Wang, Jiang-Hui
Wong, Vickie H.Y.
Bui, Bang V.
Liu, Guei-Sheung
Venue: Molecular Therapy
Publisher: Elsevier BV
Published: 2020-10
Volume: 28
Issue: 10
Pages: 2120-2138
ISSN: 1525-0016 (ISSN-L)
DOI: 10.1016/j.ymthe.2020.06.029
PMC: PMC7544979
OpenAlex: W3038555270
Cited by: 74
References: 235
Citations by year: 2026: 8 · 2025: 12 · 2024: 15 · 2023: 13 · 2022: 14 · 2021: 10 · 2020: 2
FWCI: 1.66
Type: journal article
Language: en
Open access: hybrid
doi.org
Licence: cc-by-nc-nd
www.elsevier.com/tdm/userlicense/1.0/ (tdm)
www.elsevier.com/legal/tdmrep-license (tdm)
creativecommons.org/licenses/by-nc-nd/4.0/ (vor, 371 d)
Locations: Molecular Therapy (publishedVersion, cc-by-nc-nd)
Europe PMC (PubMed Central) (publishedVersion)
PubMed Central (publishedVersion)
PubMed (publishedVersion)
UTAS Research Repository (submittedVersion)
Figshare (submittedVersion, cc-by)
Minerva Access (University of Melbourne) (submittedVersion, cc-by-nc-nd)
Swinburne figshare (Swinburne University of Technology) (submittedVersion)
Funders: National Health and Medical Research Council (GNT1185600)
Shenzhen Institutes of Advanced Technology, Chinese Academy of Sciences (ZDSYS20190902093409851)
State Government of Victoria
Ophthalmic Research Institute of Australia
Topics: Health Sciences › Medicine › Ophthalmology › Retinal Diseases and Treatments
Health Sciences › Medicine › Ophthalmology › Retinal Development and Disorders
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Angiogenesis and VEGF in Cancer
Keywords: gene therapy, neovascularization, ocular angiogenesis, neovascular age-related macular degeneration, diabetic retinopathy, anti-VEGF therapy, vascular endothelial growth factor, angiopoietins, platelet-derived growth factor, RNA interference, CRISPR gene editing
MeSH terms: Gene Editing, Animals, Clinical Trials as Topic, Disease Susceptibility, Eye Diseases, Humans, Neovascularization, Pathologic, Platelet-Derived Growth Factor, Genetic Therapy, Treatment Outcome, Disease Management, Vascular Endothelial Growth Factor A, CRISPR-Cas Systems

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Gene Therapy Intervention in Neovascular Eye Disease: A Recent Update

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