ArticleHuman gene therapy2011
Systemic adeno-associated virus-mediated gene therapy preserves retinal ganglion cells and visual function in DBA/2J glaucomatous mice.
Article in Human gene therapy, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 35 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
35 citing papers in PubMed, 51 citations in OpenAlex.
- Intramuscular delivery of recombinant AAV expressing EpoR76E improves memory and is neuroprotective in 5xFAD transgenics.Research square · 2025Article
- Gene Therapy Targeting GD3 Synthase Protects Against MPTP-Induced Parkinsonism and Executive Dysfunction.The European journal of neuroscience · 2025Article
- Exploring dysfunctional barrier phenotypes associated with glaucoma using a human pluripotent stem cell-based model of the neurovascular unit.Fluids and barriers of the CNS · 2024Article
- Long-term HIF-1α stabilization reduces respiration, promotes mitophagy, and results in retinal cell death.Scientific reports · 2023Article
- Intraocular Sustained Release of EPO-R76E Mitigates Glaucoma Pathogenesis by Activating the NRF2/ARE Pathway.Antioxidants (Basel, Switzerland) · 2023Article
- Erythropoietin in Glaucoma: From Mechanism to Therapy.International journal of molecular sciences · 2023Review
- Topical Administration of a Nanoformulation of Chitosan-Hyaluronic Acid-Epoetin Beta in a Rat Model of Glaucoma.Pharmaceuticals (Basel, Switzerland) · 2023Article
- The Latest Evidence of Erythropoietin in the Treatment of Glaucoma.International journal of molecular sciences · 2022Review
- Retinal oxidative stress activates the NRF2/ARE pathway: An early endogenous protective response to ocular hypertension.Redox biology · 2021Article
- Erythropoietin Gene Therapy Delays Retinal Degeneration Resulting from Oxidative Stress in the Retinal Pigment Epithelium.Antioxidants (Basel, Switzerland) · 2021Article
- Use of Gene Therapy in Retinal Ganglion Cell Neuroprotection: Current Concepts and Future Directions.Biomolecules · 2021Review
- Looking into the future: Gene and cell therapies for glaucoma.Veterinary ophthalmology · 2021Review
- Microsphere antioxidant and sustained erythropoietin-R76E release functions cooperate to reduce traumatic optic neuropathy.Journal of controlled release : official journal of the Controlled Release Society · 2021Article
- Structural and Functional Rescue of Chronic Metabolically Stressed Optic Nerves through Respiration.The Journal of neuroscience : the official journal of the Society for Neuroscience · 2018Article
- Effect of the Notch signaling pathway on retinal ganglion cells and its neuroprotection in rats with acute ocular hypertension.International journal of ophthalmology · 2018Article
- Neuroprotection by eIF2α-CHOP inhibition and XBP-1 activation in EAE/optic neuritiss.Cell death & disease · 2017Article
- Neuroglobin Can Prevent or Reverse Glaucomatous Progression in DBA/2J Mice.Molecular therapy. Methods & clinical development · 2017Article
- Hypoxia-Inducible Factor-1α Target Genes Contribute to Retinal Neuroprotection.Frontiers in cellular neuroscience · 2017Review
- Erythropoietin either Prevents or Exacerbates Retinal Damage from Eye Trauma Depending on Treatment Timing.Optometry and vision science : official publication of the American Academy of Optometry · 2017Article
- Overexpression of Soluble Fas Ligand following Adeno-Associated Virus Gene Therapy Prevents Retinal Ganglion Cell Death in Chronic and Acute Murine Models of Glaucoma.Journal of immunology (Baltimore, Md. : 1950) · 2016Article
Corrections and comments
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Authors and funding
4 authors at 1 institution in 1 country.
Funding
Abstract
A slow progressive death of neurons is the hallmark of neurodegenerative diseases, such as glaucoma. A therapeutic candidate, erythropoietin (EPO), has shown promise in many models of these diseases; however, it also causes polycythemia, a potentially lethal side effect. We have developed a novel mutant form of EPO that is neuroprotective but no longer erythropoietic by altering a single amino acid (arginine to glutamate at position 76; R76E). We hypothesized that a single intramuscular injection of recombinant adeno-associated virus carrying EpoR76E (rAAV2/5.CMV.EpoR76E) would protect retinal ganglion cells in a mouse model of glaucoma without inducing polycythemia. This systemic treatment not only protected the retinal ganglion cell somata located within the retina; it also preserved axonal projections within the optic nerve, while maintaining the hematocrit within normal limits. The rescued retinal ganglion cells retained their visual function demonstrated by flash visual evoked potentials. To our knowledge, this is the first demonstration of a therapy that protects neurons from death and prevents loss of visual function from the slow neurodegenerative effects of glaucoma. Because of its broad range of cellular targets, EpoR76E is likely to be successful in treating other neurodegenerative diseases as well.
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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.