ArticleStem cell research & therapy2021
Transplantation of retinal pigment epithelium and photoreceptors generated concomitantly via small molecule-mediated differentiation rescues visual function in rodent models of retinal degeneration.
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 29 papers.
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Who cites it
29 citing papers in PubMed, 39 citations in OpenAlex.
- A fatty acid amide activates myeloid cells and improves neurovascular outcomes in retinal degeneration.Nature neuroscience · 2026Article
- Application of human induced pluripotent stem cells for tissue modeling and therapy: are we on track?Stem cells (Dayton, Ohio) · 2026Review
- 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
- Efficient derivation of hiPSC-derived photoreceptor precursor cells and their neuroprotective effects in retinal degeneration.iScience · 2025Article
- Chemical reprogramming of fibroblasts into retinal pigment epithelium cells for vision restoration.Nature communications · 2025Article
- Animal models for the evaluation of retinal stem cell therapies.Progress in retinal and eye research · 2025Review
- Allogeneic RPE cell suspension manufactured at scale demonstrating preclinical safety and efficacy led to IND approval.NPJ Regenerative medicine · 2025Article
- Review
- Differentiation of human induced pluripotent stem cells into retinal pigment epithelium cells during culture on peptide-grafted hydrogels.Regenerative biomaterials · 2025Article
- Using Small Molecules to Reprogram RPE Cells in Regenerative Medicine for Degenerative Eye Disease.Cells · 2024Review
- Cell Therapy for Retinal Degenerative Diseases: Progress and Prospects.Pharmaceutics · 2024Review
- Review
- Tissue Engineering and Ophthalmology.Turkish journal of ophthalmology · 2024Review
- Generation and characterization of retinal pigment epithelium from patient iPSC line to model oculocutaneous albinism (OCA)1A disease.Journal of biosciences · 2024Article
- Effects of ECM protein-coated surfaces on the generation of retinal pigment epithelium cells differentiated from human pluripotent stem cells.Regenerative biomaterials · 2024Article
- Comparison of retinal degeneration treatment with four types of different mesenchymal stem cells, human induced pluripotent stem cells and RPE cells in a rat retinal degeneration model.Journal of translational medicine · 2023Article
- Stem cell therapy in retinal diseases.Neural regeneration research · 2023Review
- Retinitis Pigmentosa: Novel Therapeutic Targets and Drug Development.Pharmaceutics · 2023Review
- Novel Approaches in the Drug Development and Delivery Systems for Age-Related Macular Degeneration.Life (Basel, Switzerland) · 2023Review
- An improved protocol for generation and characterization of human-induced pluripotent stem cell-derived retinal pigment epithelium cells.STAR protocols · 2022Article
Corrections and comments
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Authors and funding
8 authors at 4 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundAge-related macular degeneration (AMD) is a result of degeneration/damage of the retinal pigment epithelium (RPE) while retinitis pigmentosa (RP), an inherited early-onset disease, results from premature loss of photoreceptors. A promising therapeutic approach for both is the replacement of lost/damaged cells with human induced pluripotent stem cell (hiPSC)-derived retinal cells.
methodsThe aim of this study was to investigate the in vivo functionality of RPE and photoreceptor progenitor (PRP) cells derived from a clinical-grade hiPSC line through a unified protocol. De novo-generated RPE and PRP were characterized extensively to validate their identity, purity, and potency.
resultsRPE expressed tight junction proteins, showed pigmentation and ciliation, and secreted polarization-related factors vascular endothelial growth factor (VEGF) and pigment epithelium-derived factor (PEDF). PRP expressed neural retina proteins and cone and rod markers, and responded to KCl-induced polarization. Transcriptomic analysis demonstrated an increase in the expression of mature retinal tissue-specific genes coupled with concomitant downregulation of genes from undesired lineages. RPE transplantation rescued visual function in RCS rats shown via optokinetic tracking and photoreceptor rescue. PRP transplantation improved light perception in NOD.SCID-rd1 mice, and positive electroretinography signals indicated functional photoreceptor activity in the host's outer nuclear layer. Graft survival and integration were confirmed using immunohistochemistry, and no animals showed teratoma formation or any kind of ectopic growth in the eye.
conclusionsTo our knowledge, this is the first demonstration of a unified, scalable, and GMP-adaptable protocol indicating strong animal efficacy and safety data with hiPSC-derived RPE and PRP cells. These findings provide robust proof-of-principle results for IND-enabling studies to test these potential regenerative cell therapies in patients.
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