ArticleAnnals of translational medicine2021
Transplantation of GMP-grade human iPSC-derived retinal pigment epithelial cells in rodent model: the first pre-clinical study for safety and efficacy in China.
Article in Annals of translational medicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers.
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20 citing papers in PubMed, 46 citations in OpenAlex.
- Retinal Pigment Epithelium Extracellular Vesicles Induce Microglia Polarization in MERTK-Associated Retinal Degeneration.Investigative ophthalmology & visual science · 2025Article
- Stem cell therapy for retinal pigment epithelium disorders.World journal of stem cells · 2025Review
- 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
- Advances in the Study of Age-Related Macular Degeneration Based on Cell or Cell-Biomaterial Scaffolds.Bioengineering (Basel, Switzerland) · 2025Review
- Clinical translation of human iPSC technologies: advances, safety concerns, and future directions.Frontiers in cell and developmental biology · 2025Review
- Atrophic Macular Degeneration and Stem Cell Therapy: A Clinical Review.Advances in experimental medicine and biology · 2025Review
- Patient-derived induced pluripotent stem cells with aWorld journal of stem cells · 2024Article
- Tissue engineering RPE sheet derived from hiPSC-RPE cell spheroids supplemented with Y-27632 and RepSox.Journal of biological engineering · 2024Article
- Human and Pig Pluripotent Stem Cells: From Cellular Products to Organogenesis and Beyond.Cells · 2023Review
- Progress in Stem Cells-Based Replacement Therapy for Retinal Pigment Epithelium: In Vitro Differentiation to In Vivo Delivery.Stem cells translational medicine · 2023Review
- Single-cell transcriptomics reveals maturation of transplanted stem cell-derived retinal pigment epithelial cells toward native state.Proceedings of the National Academy of Sciences of the United States of America · 2023Article
- Advancing cell therapy for neurodegenerative diseases.Cell stem cell · 2023Review
- Generation and Differentiation of Induced Pluripotent Stem Cells from Mononuclear Cells in An Age-Related Macular Degeneration Patient.Cell journal · 2022Article
- Advances in cell therapies using stem cells/progenitors as a novel approach for neurovascular repair of the diabetic retina.Stem cell research & therapy · 2022Review
- Global spectrum ofFrontiers in aging neuroscience · 2022Article
- Review
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- Efficient differentiation of human embryonic stem cells to retinal pigment epithelium under defined conditions.Stem cell research & therapy · 2021Article
- Improvement of human embryonic stem cell-derived retinal pigment epithelium cell adhesion, maturation, and function through coating with truncated recombinant human vitronectin.International journal of ophthalmology · 2021Article
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Authors and funding
9 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundAge-related macular degeneration (AMD) is the leading cause of blindness in the elderly due in large part to age-dependent atrophy of retinal pigment epithelium (RPE) cells. RPE cells form a monolayer located between the choroid and the outer segments of photoreceptors, playing multifarious roles in maintenance of visual function. Allogeneically induced pluripotent stem cell-derived RPE (iPSC-RPE or iRPE) has become a potential approach for providing an abundant source of donors for clinical cell products. Transplantation of iRPE has been proven effective in rescuing impaired retinas in Royal College of Surgeons (RCS) rats after approximately 5 to 6 weeks. Here, we explore the long-term (19 weeks) safety and efficacy of human iRPE cell transplantation in pre-clinical animal models.
methodsThe expression of human RPE-specific markers in iRPE cells was determined using immunofluorescence staining. For the proliferative test, Ki-67 expression was also verified by immunofluorescence and flow cytometric analysis. Then, iRPE cells were transplanted into the subretinal space of immune-deficient NOD/SCID/IL-2Rgc
resultsHuman iRPE cells expressed native RPE-specific markers, such as microphthalmia-associated transcription factor (MiTF), retinal pigment epithelium-specific 65-kDa protein (RPE65) and tight-junction associated structural protein (ZO-1), and their proliferative capacity (Ki-67 expression) was poor after 25 days of induction. A tumorigenicity test revealed no tumor formation or abnormal proliferation in the immunodeficient mice after subretinal injection of 5×10
conclusionsIn the present study, we provided further evidence for the use of human iRPE transplantation to treat retinal degenerative disease in pre-clinical animal models. Therefore, we consider human iRPE cells a promising source of cell replacement therapy for AMD.
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