Emerging strategies in corneal Regeneration: The role of induced pluripotent stem cell-based therapies

Sep 10, 2025Experimental eye research

New approaches to cornea repair using stem cells made from adult cells

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Regenerative Health on OpenScience ↗PubMed ↗DOI ↗

Abstract

Induced pluripotent stem cells (iPSCs) may provide a promising approach to generating corneal-like cells for corneal regeneration.

Corneal opacity is a leading cause of global blindness, with traditional transplantation methods facing significant limitations.
Regenerative strategies aim to restore the integrity of all three corneal layers: epithelium, stroma, and endothelium.
Recent clinical trials using iPSC-derived corneal cells show early safety and efficacy outcomes.
Challenges associated with iPSC therapies include tumor formation, immune responses, and cell variability.
Potential solutions to these challenges include creating HLA-matched iPSC banks and employing gene editing techniques.

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Corneal opacity remains a leading cause of global blindness, yet conventional corneal transplantation is constrained by donor scarcity, surgical limitations, and suboptimal long-term outcomes. In response, regenerative strategies are advancing to restore structural and functional integrity across all three corneal layers-epithelium, stroma, and endothelium-through cell-based and bioengineered therapies. Among these, induced pluripotent stem cells (iPSCs) have emerged as a versatile and scalable source capable of generating corneal-like cells under defined, xeno-free conditions. This review provides a concise summary of recent clinical progress in corneal cell therapy and tissue engineering, while placing major emphasis on systematically updating preclinical advances in iPSC-based corneal regeneration. Additionally, we highlight the only two first-in-human trials employing iPSC-derived corneal cells, which demonstrate encouraging early safety and efficacy outcomes, yet underscore critical translational challenges including tumorigenicity, immunogenicity, and heterogeneity. Emerging solutions-such as HLA-matched iPSC banks, gene editing, and naïve-state reprogramming-are discussed as key strategies to enhance clinical readiness and scalability.

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Emerging strategies in corneal Regeneration: The role of induced pluripotent stem cell-based therapies

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