ArticlePloS one2011
Efficacious and safe tissue-selective controlled gene therapy approaches for the cornea.
Article in PloS one, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
19 citing papers in PubMed, 27 citations in OpenAlex.
- Topical application of Cas9 ribonucleoproteins inhibits corneal neovascularization in a mouse model of alkali burn injury.Gene therapy · 2026Article
- 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
- Review
- AAV for Gene Therapy in Ocular Diseases: Progress and Prospects.Research (Washington, D.C.) · 2023Review
- Corneal stromal repair and regeneration.Progress in retinal and eye research · 2022Review
- Long-Term Safety and Tolerability of BMP7 and HGF Gene Overexpression in Rabbit Cornea.Translational vision science & technology · 2021Article
- Six-Month In Vivo Safety Profiling of Topical Ocular AAV5-Decorin Gene Transfer.Translational vision science & technology · 2021Article
- Novel insights into gene therapy in the cornea.Experimental eye research · 2021Review
- Adeno-Associated Virus Mediated Gene Therapy for Corneal Diseases.Pharmaceutics · 2020Review
- Preparation and Administration of Adeno-associated Virus Vectors for Corneal Gene Delivery.Methods in molecular biology (Clifton, N.J.) · 2020Article
- MMP-9 Downregulation with Lipid Nanoparticles for Inhibiting Corneal Neovascularization by Gene Silencing.Nanomaterials (Basel, Switzerland) · 2019Article
- Serotype survey of AAV gene delivery via subconjunctival injection in mice.Gene therapy · 2018Article
- Article
- Gene editing for corneal disease management.World journal of translational medicine · 2016Article
- Corneal gene therapy: basic science and translational perspective.The ocular surface · 2013Review
- BMP7 gene transfer via gold nanoparticles into stroma inhibits corneal fibrosis in vivo.PloS one · 2013Article
- Article
- Significant inhibition of corneal scarring in vivo with tissue-selective, targeted AAV5 decorin gene therapy.Investigative ophthalmology & visual science · 2011Article
- Linear Polyethylenimine-DNA Nanoconstruct for Corneal Gene Delivery.Journal of ocular pharmacology and therapeutics : the official journal of the Association for Ocular Pharmacology and TherapeuticsArticle
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 4 institutions in 1 country.
Funding
Abstract
Untargeted and uncontrolled gene delivery is a major cause of gene therapy failure. This study aimed to define efficient and safe tissue-selective targeted gene therapy approaches for delivering genes into keratocytes of the cornea in vivo using a normal or diseased rabbit model. New Zealand White rabbits, adeno-associated virus serotype 5 (AAV5), and a minimally invasive hair-dryer based vector-delivery technique were used. Fifty microliters of AAV5 titer (6.5×10(12) vg/ml) expressing green fluorescent protein gene (GFP) was topically applied onto normal or diseased (fibrotic or neovascularized) rabbit corneas for 2-minutes with a custom vector-delivery technique. Corneal fibrosis and neovascularization in rabbit eyes were induced with photorefractive keratectomy using excimer laser and VEGF (630 ng) using micropocket assay, respectively. Slit-lamp biomicroscopy and immunocytochemistry were used to confirm fibrosis and neovascularization in rabbit corneas. The levels, location and duration of delivered-GFP gene expression in the rabbit stroma were measured with immunocytochemistry and/or western blotting. Slot-blot measured delivered-GFP gene copy number. Confocal microscopy performed in whole-mounts of cornea and thick corneal sections determined geometric and spatial localization of delivered-GFP in three-dimensional arrangement. AAV5 toxicity and safety were evaluated with clinical eye exam, stereomicroscopy, slit-lamp biomicroscopy, and H&E staining. A single 2-minute AAV5 topical application via custom delivery-technique efficiently and selectively transduced keratocytes in the anterior stroma of normal and diseased rabbit corneas as evident from immunocytochemistry and confocal microscopy. Transgene expression was first detected at day 3, peaked at day 7, and was maintained up to 16 weeks (longest tested time point). Clinical and slit-lamp eye examination in live rabbits and H&E staining did not reveal any significant changes between AAV5-treated and untreated control corneas. These findings suggest that defined gene therapy approaches are safe for delivering genes into keratocytes in vivo and has potential for treating corneal disorders in human patients.
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