Viral-vector-delivered anti-angiogenic therapies to the eye
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Abstract
Pathological vessel growth harms vision and may finally lead to vision loss. Anti-angiogenic gene therapy with viral vectors for ocular neovascularization has shown great promise in preclinical studies. Most of the studies have been conducted with different adeno-associated serotype vectors. In addition, adeno- and lentivirus vectors have been used. Therapy has been targeted towards blocking vascular endothelial growth factors or other pro-angiogenic factors. Clinical trials of intraocular gene therapy for neovascularization have shown the treatment to be safe without severe adverse events or systemic effects. Nevertheless, clinical studies have not proceeded further than Phase 2 trials.
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IVT injection potentially exposes the entire retina to gene therapy.
“IVT injection potentially exposes the entire retina to gene therapy (Koponen et al., 2021; Puranen et al., 2022), whereas delivery to subretinal space allows direct contact of the vector with photoreceptors and the RPE layers (Irigoyen et al., 2022).”
From AAV2 and AAV9 tropism and transgene expression in the mouse eye and major tissues after intravitreal and subretinal delivery (2023) · cites this paper as Koponen et al., 2021
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- Authors
- Koponen, S., Kokki, E., Kinnunen, K., & Ylä-Herttuala, S.
- Affiliations
- University of Eastern Finland (FI)
- Kuopio University Hospital (FI)
- A.I. Virtanen Institute for Molecular Sciences, University of Eastern Finland, P.O. Box 1627, 70211 Kuopio, Finland
- Venue
- Pharmaceutics
- Publisher
- MDPI AG
- Published
- 2021-02-05
- Volume
- 13
- Issue
- 2
- Pages
- 219
- ISSN
- 1999-4923 (ISSN-L)
- PMC
- PMC7915489
- OpenAlex
- W3126173951
- Cited by
- 24
- References
- 125
- Citations by year
- 2026: 2 · 2025: 7 · 2024: 3 · 2023: 3 · 2022: 6 · 2021: 3
- FWCI
- 0.55
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Licence
- Locations
- Pharmaceutics (publishedVersion, cc-by)
- Preprints.org (acceptedVersion, cc-by)
- PubMed Central (publishedVersion, cc-by)
- PubMed (publishedVersion)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Angiogenesis and VEGF in Cancer
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Retinal Development and Disorders
- Keywords
- antiangiogenic gene therapy, viral vectors, ocular neovascularization, adeno-associated virus vectors, lentiviral vectors, vascular endothelial growth factor, vision loss, clinical trials
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