ArticlePharmaceutics2020
Transferrin Non-Viral Gene Therapy for Treatment of Retinal Degeneration.
Article in Pharmaceutics, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 14 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
An Open-label Single Ascending Dose Safety and Tolerability Clinical Trial of PST-611 in Subjects With Dry Age-related Macular Degeneration
An Open-label Multiple Dose Safety, Tolerability and Exploratory Efficacy Clinical Trial of PST-611 in Patients With Geographic Atrophy Secondary to Age-related Macular Degeneration
Who cites it
14 citing papers in PubMed, 1 synthesis or guideline pooled it, 22 citations in OpenAlex.
- Systematic review and meta-analysis of mass spectrometry proteomics applied to ocular fluids to assess potential biomarkers of age-related macular degeneration.BMC ophthalmology · 2023Pooled it
- Photoreceptor Vulnerability to Ferroptosis: Membrane Phospholipid Peroxidation, Mitochondrial Homeostasis, and RPE-Photoreceptor Coupling.Current issues in molecular biology · 2026Review
- Carnosic acid's mechanism in alleviating POAG-induced optic nerve injuryInternational journal of ophthalmology · 2026Article
- Apo-Transferrin Modified Natamycin-Loaded Liposomes Improve the Prognosis of Fungal Keratitis via Enhanced Antifungal and Anti-Inflammatory Activities.International journal of nanomedicine · 2026Article
- Ferroptosis in retinal neurodegeneration: mechanistic vulnerability, therapeutic targeting, and translational barriers.Frontiers in medicine · 2026Review
- Reactive Oxygen Species Regulation Across Retinitis Pigmentosa Animal Models: A 25-Year Systematized Review.Molecular neurobiology · 2025Review
- Transferrin is a drug candidate for the treatment of dry age-related macular degeneration (AMD).Cell death & disease · 2025Article
- Levels of Zinc, Iron, and Copper in the Aqueous Humor of Patients with Primary Glaucoma.Biomolecules · 2025Article
- Targeting ferroptosis: a novel therapeutic strategy for the treatment of retinal diseases.Frontiers in pharmacology · 2024Review
- Neuroprotective Effects of Transferrin in Experimental Glaucoma Models.International journal of molecular sciences · 2022Article
- Ocular Barriers and Their Influence on Gene Therapy Products Delivery.Pharmaceutics · 2022Review
- Iron contributes to photoreceptor degeneration and Müller glia proliferation in the zebrafish light-treated retina.Experimental eye research · 2022Article
- Review
- Insights into the Roles of the Sideroflexins/SLC56 Family in Iron Homeostasis and Iron-Sulfur Biogenesis.Biomedicines · 2021Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
9 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Dysregulation of iron metabolism is observed in animal models of retinitis pigmentosa (RP) and in patients with age-related macular degeneration (AMD), possibly contributing to oxidative damage of the retina. Transferrin (TF), an endogenous iron chelator, was proposed as a therapeutic candidate. Here, the efficacy of TF non-viral gene therapy based on the electrotransfection of pEYS611, a plasmid encoding human TF, into the ciliary muscle was evaluated in several rat models of retinal degeneration. pEYS611 administration allowed for the sustained intraocular production of TF for at least 3 and 6 months in rats and rabbits, respectively. In the photo-oxidative damage model, pEYS611 protected both retinal structure and function more efficiently than carnosic acid, a natural antioxidant, reduced microglial infiltration in the outer retina and preserved the integrity of the outer retinal barrier. pEYS611 also protected photoreceptors from N-methyl-N-nitrosourea-induced apoptosis. Finally, pEYS611 delayed structural and functional degeneration in the RCS rat model of RP while malondialdehyde (MDA) ocular content, a biomarker of oxidative stress, was decreased. The neuroprotective benefits of TF non-viral gene delivery in retinal degenerative disease models further validates iron overload as a therapeutic target and supports the continued development of pEY611 for treatment of RP and dry AMD.
Indexed as
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What OpenQuestion holds
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.