ArticleMolecular therapy : the journal of the American Society of Gene Therapy2018
Otx2-Genetically Modified Retinal Pigment Epithelial Cells Rescue Photoreceptors after Transplantation.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers, 1 of them a synthesis that pooled it.
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Who cites it
22 citing papers in PubMed, 1 synthesis or guideline pooled it, 0 citations in OpenAlex.
- A Systematic Review on Transplantation Studies of the Retinal Pigment Epithelium in Animal Models.International journal of molecular sciences · 2020Pooled it
- IND-enabling safety and efficacy of RPESC-RPE-4W, an adult RPE progenitor cell therapy for dry age-related macular degeneration.Cell reports. Medicine · 2026Article
- Combined Expression of hRdCVF and hRdCVFL Through AAV-Mediated Delivery for the Treatment of Retinitis Pigmentosa.Investigative ophthalmology & visual science · 2026Article
- GPR40 Attenuates Age-Related Macular Degeneration by Suppressing Retinal Microglial NLRP3 Inflammasome Activation Via ERK Signaling.Inflammation · 2026Article
- Article
- Retinal Pigment Epithelium Transplantation in Retinal Disease: Clinical Trial Development, Challenges, and Future Directions.Biomolecules · 2025Review
- Animal models for the evaluation of retinal stem cell therapies.Progress in retinal and eye research · 2025Review
- A single-cell chromatin accessibility dataset of human primed and naïve pluripotent stem cell-derived teratoma.Scientific data · 2024Article
- Cone-rod homeobox transcriptionally activates TCF7 to promote the proliferation of retinal pigment epithelial and retinoblastoma cellsInternational journal of ophthalmology · 2024Article
- Review
- Progress of iPS cell-based transplantation therapy for retinal diseases.Japanese journal of ophthalmology · 2023Review
- OTX2 signaling in retinal dysfunction, degeneration and regeneration.Neural regeneration research · 2021Article
- A universal protocol for isolating retinal ON bipolar cells across species via fluorescence-activated cell sorting.Molecular therapy. Methods & clinical development · 2021Article
- Viral delivery of multiple miRNAs promotes retinal ganglion cell survival and functional preservation after optic nerve crush injury.Experimental eye research · 2020Article
- Vitamin C- and Valproic Acid-Induced Fetal RPE Stem-like Cells Recover Retinal Degeneration via Regulating SOX2.Molecular therapy : the journal of the American Society of Gene Therapy · 2020Article
- Metabolic and Redox Signaling of the Nucleoredoxin-Like-1 Gene for the Treatment of Genetic Retinal Diseases.International journal of molecular sciences · 2020Review
- Activating Transcription Factor 3 (ATF3) Protects Retinal Ganglion Cells and Promotes Functional Preservation After Optic Nerve Crush.Investigative ophthalmology & visual science · 2020Article
- Mechanisms Underlying the Visual Benefit of Cell Transplantation for the Treatment of Retinal Degenerations.International journal of molecular sciences · 2019Review
- A novel xeno-free culture system for human retinal pigment epithelium cells.International journal of ophthalmology · 2019Article
- Blue Autofluorescence Fundus Imaging for Monitoring Retinal Degeneration in Royal College of Surgeons Rats.Translational vision science & technology · 2019Article
Corrections and comments
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Authors and funding
16 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Inherited retinal degenerations are blinding diseases characterized by the loss of photoreceptors. Their extreme genetic heterogeneity complicates treatment by gene therapy. This has motivated broader strategies for transplantation of healthy retinal pigmented epithelium to protect photoreceptors independently of the gene causing the disease. The limited clinical benefit for visual function reported up to now is mainly due to dedifferentiation of the transplanted cells that undergo an epithelial-mesenchymal transition. We have studied this mechanism in vitro and revealed the role of the homeogene OTX2 in preventing dedifferentiation through the regulation of target genes. We have overexpressed OTX2 in retinal pigmented epithelial cells before their transplantation in the eye of a model of retinitis pigmentosa carrying a mutation in Mertk, a gene specifically expressed by retinal pigmented epithelial cells. OTX2 increases significantly the protection of photoreceptors as seen by histological and functional analyses. We observed that the beneficial effect of OTX2 is non-cell autonomous, and it is at least partly mediated by unidentified trophic factors. Transplantation of OTX2-genetically modified cells may be medically effective for other retinal diseases involving the retinal pigmented epithelium as age-related macular degeneration.
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