The DOI matches its Crossref record; 232 of the 235 references it cites were found in a registry or an index.
Gene Therapy Intervention in Neovascular Eye Disease: A Recent Update
Open accessAbstract
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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“Intravitreal administration of anti-VEGF drugs is the approved front-line treatment modality for these patients (17).”
From Characterization of RNA editing and gene therapy with a compact CRISPR-Cas13 in the retina (2024) · cites this paper as [17]
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2 retracted
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RetractedMultiplex Genome Engineering Using CRISPR/Cas Systems
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Self-citationSearch-and-replace genome editing without double-strand breaks or donor DNA
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Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System
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Han, H., Li, S., Xu, M., Zhong, Y., Fan, W., Xu, J., Zhou, T., Ji, J., Ye, J., & Yao, K. (2023) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2023.114770 · Cited by 341
Dual-AAV delivering split prime editor system for in vivo genome editing
Zhi, S., Chen, Y., Wu, G., Wen, J., Wu, J., Liu, Q., Li, Y., Kang, R., Hu, S., Wang, J., et al. (2022) · Molecular Therapy · doi:10.1016/j.ymthe.2021.07.011 · Cited by 174
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Available Therapeutic Options for Corneal Neovascularization: A Review
Drzyzga, Ł. R., Śpiewak, D., Dorecka, M., & Wyględowska‐Promieńska, D. (2024) · International Journal of Molecular Sciences · doi:10.3390/ijms25105479 · Cited by 77
Review: The Development of Risk Factors and Cytokines in Retinal Vein Occlusion
Tang, Y. P., Cheng, Y. F., Wang, S., Wang, Y., Liu, P., & Wu, H. (2022) · Frontiers in Medicine · doi:10.3389/fmed.2022.910600 · Cited by 63
Metadata
- 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
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2020-10
- Volume
- 28
- Issue
- 10
- Pages
- 2120-2138
- ISSN
- 1525-0016 (ISSN-L)
- 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
- 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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