Safety and efficacy of human ESC-derived corneal endothelial cells for corneal endothelial dysfunction

Here we defined an adapted differentiation protocol to generate induced corneal endothelial cells (iCECs) consistently and efficiently from clinical-grade human embryonic stem cells (hESCs) with xeno-free medium and manufactured cryopreserved iCECs. Cells express high levels of typical CECs markers...

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Bibliographic Details
Published in:Cell & bioscience 2023-11, Vol.13 (1), p.1-201, Article 201
Main Authors: Yu, Juan, Yu, Nianye, Tian, Yao, Fang, Yifan, An, Bin, Feng, Guihai, Wu, Jun, Wang, Liu, Hao, Jie, Wang, Liqiang, Zhou, Qi, Li, Wei, Wang, Yukai, Hu, Baoyang
Format: Article
Language:English
Subjects:
Adenosine triphosphatase
Analysis
Animal models
Cell therapy
Computer software industry
Cornea
Corneal endothelial dysfunction
Corneal transplantation
Cryopreservation
Edema
Embryo cells
Embryonic stem cells
Endothelial cells
Endothelium
Gene expression
Health aspects
Human embryonic stem cells (hESCs)
Immunodeficiency
Induced corneal endothelial cells (iCECs)
Pluripotency
Quality control
Regenerative medicine
Stem cells
Transplantation
Tumorigenicity
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Summary:Here we defined an adapted differentiation protocol to generate induced corneal endothelial cells (iCECs) consistently and efficiently from clinical-grade human embryonic stem cells (hESCs) with xeno-free medium and manufactured cryopreserved iCECs. Cells express high levels of typical CECs markers and exhibit transendothelial potential properties in vitro typical of iCECs. After rigorous quality control measures, cells meeting all release criteria were available for in vivo studies. We found that there was no overgrowth or tumorigenicity of grafts in immunodeficient mice. After grafting into rabbit models, the surviving iCECs ameliorated edema and recovered corneal opacity. Our work provides an efficient approach for generating iCECs and demonstrates the safety and efficacy of iCECs in disease modeling. Therefore, clinical-grade iCECs are a reliable source for future clinical treatment of corneal endothelial dysfunction.
ISSN:2045-3701
2045-3701
DOI:10.1186/s13578-023-01145-w