ArticleScientific reports2018
Development of an inducible anti-VEGF rAAV gene therapy strategy for the treatment of wet AMD.
Article in Scientific reports, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 39 papers, 1 of them a synthesis that pooled it.
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
39 citing papers in PubMed, 1 synthesis or guideline pooled it, 60 citations in OpenAlex.
- Recombinant Adeno-Associated Viral Vectors (rAAV)-Vector Elements in Ocular Gene Therapy Clinical Trials and Transgene Expression and Bioactivity Assays.International journal of molecular sciences · 2020Pooled it
- Mammalian therapeutic riboswitches: Engineering and custom ligands.Molecular therapy. Nucleic acids · 2026Review
- Molecular Determinants of Intravitreal Anti-VEGF Durability: Drug Architecture, Intraocular Pharmacokinetics, Target Biology and Treatment Resistance.International journal of molecular sciences · 2026Review
- The Molecular Basis of Ocular Aging: Mechanisms, Pathologies, and Emerging Therapeutics.Investigative ophthalmology & visual science · 2026Review
- Comparative Analysis of rAAV Production from Plasmid-Encoded Versus Chromosomally Integrated rAAV Transgene in HEK293 Cells.International journal of molecular sciences · 2026Article
- Article
- Suboptimal Responses to Anti-VEGF in Retinal Neurovascular Diseases: Linking Aging and Alternative Angioinflammatory Pathways.Investigative ophthalmology & visual science · 2026Review
- Vascular regeneration and blood flow recovery in glaucoma.Frontiers in cell and developmental biology · 2026Review
- Ultrasound Control of Gene Expression in Human iPSCs via Heat Shock Promoters.Biotechnology and bioengineering · 2025Article
- Potential Therapeutic Actions of Flavonoids Present in Propolis That Modulate Vascular Endothelial Growth Factor Signaling to Regulate Angiogenesis.Phytotherapy research : PTR · 2025Review
- Dynamic activation of rAAV transgene expression by a small molecule that recruits endogenous transcriptional machinery.Nucleic acids research · 2025Article
- Suppression of matrigel-induced choroidal neovascularization by AAV delivery of a novel anti-Scg3 antibody.Gene therapy · 2024Article
- Optimization of Exon-Skipping Riboswitches and Their Applications to Control Mammalian Cell Fate.ACS synthetic biology · 2024Article
- Cooperative binding of bivalent ligands yields new insights into the guanidine-II riboswitch.NAR genomics and bioinformatics · 2024Article
- Retinal Vein Occlusion-Background Knowledge and Foreground Knowledge Prospects-A Review.Journal of clinical medicine · 2024Review
- An adeno-associated virus variant enabling efficient ocular-directed gene delivery across species.Nature communications · 2024Article
- RNA-based controllers for engineering gene and cell therapies.Current opinion in biotechnology · 2024Review
- Riboswitch-controlled IL-12 gene therapy reduces hepatocellular cancer in mice.Frontiers in immunology · 2024Article
- A comparative survey of the influence of small self-cleaving ribozymes on gene expression in human cell culture.RNA biology · 2024Article
- Anti-Scg3 Gene Therapy to Treat Choroidal Neovascularization in Mice.Biomedicines · 2023Article
Corrections and comments
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Authors and funding
4 authors at 2 institutions in 2 countries.
Funding
Abstract
Vascular endothelial growth factor (VEGF) is a key mediator in the development and progression of choroidal neovascularization (CNV) in patients with wet age-related macular degeneration (AMD). As a consequence, current treatment strategies typically focus on the administration of anti-VEGF agents, such as Aflibercept (Eylea), that inhibit VEGF function. While this approach is largely successful at counteracting CNV progression, the treatment can require repetitive (i.e. monthly) intravitreal injections of the anti-VEGF agent throughout the patient's lifetime, imposing a substantial financial and medical burden on the patient. Moreover, repetitive injection of anti-VEGF agents over a period of years may encourage progression of retinal and choroidal atrophy in patients with AMD, leading to a decrease in visual acuity. Herein, we have developed a single-injection recombinant adeno-associated virus (rAAV)-based gene therapy treatment for wet AMD that prevents CNV formation through inducible over-expression of Eylea. First, we demonstrate that by incorporating riboswitch elements into the rAAV expression cassette allows protein expression levels to be modulated in vivo through oral supplementation on an activating ligand (e.g. tetracycline). We subsequently utilized this technology to modulate the intraocular concentration of Eylea following rAAV delivery, leading to nearly complete (p = 0.0008) inhibition of clinically significant CNV lesions in an established mouse model of wet AMD. The results shown in this study pave the way for the development of a personalized gene therapy strategy for the treatment of wet AMD that is substantially less invasive and more clinically adaptable than the current treatment paradigm of repetitive bolus injections of anti-VEGF agents.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.