ArticleInvestigative ophthalmology & visual science2010
Self-complementary AAV virus (scAAV) safe and long-term gene transfer in the trabecular meshwork of living rats and monkeys.
Article in Investigative ophthalmology & visual science, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 61 papers.
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Who cites it
61 citing papers in PubMed, 95 citations in OpenAlex.
- VEGFA-Positive Macrophages Regulate Aqueous Humor Outflow in Aged Mice and Humans.bioRxiv : the preprint server for biology · 2026Article
- AAV-IKV mediated expression of decorin inhibits EMT and fibrosis in a murine model of Glaucoma and AAV-IKV transduction in Non-Human Primates.Scientific reports · 2025Article
- Emerging strategies targeting genes and cells in glaucoma.Vision research · 2025Review
- Ocular toxicity, distribution, and shedding of intravitreal AAV-eqIL-10 in horses.Molecular therapy. Methods & clinical development · 2024Article
- Gene therapy for glaucoma: Targeting key mechanisms.Vision research · 2024Review
- Schlemm's canal-selective Tie2/TEK knockdown induces sustained ocular hypertension in adult mice.Experimental eye research · 2024Article
- Lentiviral mediated delivery of CRISPR/Cas9 reduces intraocular pressure in a mouse model of myocilin glaucoma.Scientific reports · 2024Article
- Sustained and Efficient Delivery of Antivascular Endothelial Growth Factor by the Adeno-associated Virus for the Treatment of Corneal Neovascularization: An Outlook for Its Clinical Translation.Journal of ophthalmology · 2024Review
- Lentiviral mediated delivery of CRISPR/Cas9 reduces intraocular pressure in a mouse model of myocilin glaucoma.Research square · 2023Article
- Evaluation of trabecular meshwork-specific promotersInternational journal of ophthalmology · 2023Article
- AAV for Gene Therapy in Ocular Diseases: Progress and Prospects.Research (Washington, D.C.) · 2023Review
- Adeno-associated virus vectors for retinal gene therapy in basic research and clinical studies.Frontiers in medicine · 2023Review
- Ocular Drug Delivery: Advancements and Innovations.Pharmaceutics · 2022Review
- A Novel Mouse Model of TGFβ2-Induced Ocular Hypertension Using Lentiviral Gene Delivery.International journal of molecular sciences · 2022Article
- Transgene expression of Stanniocalcin-1 provides sustained intraocular pressure reduction by increasing outflow facility.PloS one · 2022Article
- Gene Therapy in the Anterior Eye Segment.Current gene therapy · 2022Review
- Looking into the future: Gene and cell therapies for glaucoma.Veterinary ophthalmology · 2021Review
- Novel insights into gene therapy in the cornea.Experimental eye research · 2021Review
- Adeno-Associated Virus Mediated Gene Therapy for Corneal Diseases.Pharmaceutics · 2020Review
- Deconstructing aqueous humor outflow - The last 50 years.Experimental eye research · 2020Review
1 more citing papers are in PubMed but not listed here.
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9 authors at 4 institutions in 2 countries.
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Abstract
purposeAAV vectors produce stable transgene expression and elicit low immune response in many tissues. AAVs have been the vectors of choice for gene therapy for the eye, in particular the retina. scAAVs are modified AAVs that bypass the required second-strand DNA synthesis to achieve transcription of the transgene. The goal was to investigate the ability of AAV vectors to induce long-term, safe delivery of transgenes to the trabecular meshwork of living animals.
methodsSingle doses of AAV2.GFP and AAV2.RGD.GFP/Ad5.LacZ were injected intracamerally (IC) into rats (n = 28 eyes). A single dose of scAAV.GFP was IC-injected into rats (n = 72 eyes) and cynomolgus monkeys (n = 3). GFP expression was evaluated by fluorescence, immunohistochemistry, and noninvasive gonioscopy. Intraocular pressure (IOP) was measured with calibrated tonometer (rats) and Goldmann tonometer (monkeys). Differential expression of scAAV-infected human trabecular meshwork cells (HTM) was determined by microarrays. Humoral and cell-mediated immune responses were evaluated by ELISA and peripheral blood proliferation assays.
resultsNo GFP transduction was observed on the anterior segment tissues of AAV-injected rats up to 27 days after injection. In contrast, scAAV2 transduced the trabecular meshwork very efficiently, with a fast onset (4 days). Eyes remained clear and no adverse effects were observed. Transgene expression lasted >3.5 months in rats and >2.35 years in monkeys.
conclusionsThe scAAV viral vector provides prolonged and safe transduction in the trabecular meshwork of rats and monkeys. The stable expression and safe properties of this vector could facilitate the development of trabecular meshwork drugs for gene therapy for glaucoma.
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