ArticleStem cell research & therapy2021
cGMP-grade human iPSC-derived retinal photoreceptor precursor cells rescue cone photoreceptor damage in non-human primates.
Article in Stem cell research & therapy, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 papers, 1 of them a synthesis that pooled it.
What it found
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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.
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Who cites it
26 citing papers in PubMed, 1 synthesis or guideline pooled it, 32 citations in OpenAlex.
- Large animal model species in pluripotent stem cell therapy research and development for retinal diseases: a systematic review.Frontiers in ophthalmology · 2024Pooled it
- Efficient derivation of hiPSC-derived photoreceptor precursor cells and their neuroprotective effects in retinal degeneration.iScience · 2025Article
- Application of Machine Learning to Discriminate Photoreceptor Cell Species in Xenotransplanted Chimeric Retinas.Clinical and translational science · 2025Article
- Functional scaffolds design strategies for retinal repair and regeneration.Materials today. Bio · 2025Review
- Evaluating the outcomes of pluripotent stem-cell-derived photoreceptor transplantation in retinal repair.The FEBS journal · 2025Review
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- Review
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- Cellular component transfer between photoreceptor cells of the retina.Progress in retinal and eye research · 2025Review
- Cell-cell interactions between transplanted retinal organoid cells and recipient tissues.Current opinion in genetics & development · 2024Review
- Strategies for management of retinal degenerative disorders.Indian journal of ophthalmology · 2023Article
- Characteristics of Rare Inherited Retinal Dystrophies in Adaptive Optics-A Study on 53 Eyes.Diagnostics (Basel, Switzerland) · 2023Article
- Preclinical evaluation of ADVM-062, a novel intravitreal gene therapy vector for the treatment of blue cone monochromacy.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Article
- Stem cell sources and characterization in the development of cell-based products for treating retinal disease: An NEI Town Hall report.Stem cell research & therapy · 2023Article
- Automating iPSC generation to enable autologous photoreceptor cell replacement therapy.Journal of translational medicine · 2023Article
- Pan-retinal ganglion cell markers in mice, rats, and rhesus macaques.Zoological research · 2023Article
- A treatment within sight: challenges in the development of stem cell-derived photoreceptor therapies for retinal degenerative diseases.Frontiers in transplantation · 2023Review
- Therapeutic landscape for inherited ocular diseases: Current and emerging therapies.Singapore medical journal · 2023Review
- Advances in cell therapies using stem cells/progenitors as a novel approach for neurovascular repair of the diabetic retina.Stem cell research & therapy · 2022Review
- Newer therapeutic options for inherited retinal diseases: Gene and cell replacement therapy.Indian journal of ophthalmology · 2022Review
Corrections and comments
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Authors and funding
17 authors at 7 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundRetinal regenerative therapies hold great promise for the treatment of inherited retinal degenerations (IRDs). Studies in preclinical lower mammal models of IRDs have suggested visual improvement following retinal photoreceptor precursors transplantation, but there is limited evidence on the ability of these transplants to rescue retinal damage in higher mammals. The purpose of this study was to evaluate the therapeutic potential of photoreceptor precursors derived from clinically compliant induced pluripotent stem cells (iPSCs).
methodsPhotoreceptor precursors were sub-retinally transplanted into non-human primates (Macaca fascicularis). The cells were transplanted both in naïve and cobalt chloride-induced retinal degeneration models who had been receiving systemic immunosuppression for one week prior to the procedure. Optical coherence tomography, fundus autofluorescence imaging, electroretinography, ex vivo histology and immunofluorescence staining were used to evaluate retinal structure, function and survival of transplanted cells.
resultsThere were no adverse effects of iPSC-derived photoreceptor precursors on retinal structure or function in naïve NHP models, indicating good biocompatibility. In addition, photoreceptor precursors injected into cobalt chloride-induced retinal degeneration NHP models demonstrated an ability both to survive and to mature into cone photoreceptors at 3 months post-transplant. Optical coherence tomography showed restoration of retinal ellipsoid zone post-transplantation.
conclusionsThese findings demonstrate the safety and therapeutic potential of clinically compliant iPSC-derived photoreceptor precursors as a cell replacement source for future clinical trials.
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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.