Department of Immunology, Fasa University of Medical Sciences, Fasa, Iran
Abstract
Stem cell-based regenerative medicine has emerged as one of the most promising strategies for treating ocular diseases characterized by irreversible tissue damage. The eye provides a particularly attractive target for regenerative medicine because several ocular tissues contain endogenous progenitor populations, while pluripotent stem-cell technologies can generate clinically relevant corneal and retinal cell types. Among current applications, limbal stem-cell transplantation has achieved the strongest clinical translation, particularly for limbal stem cell deficiency (LSCD). In parallel, human embryonic stem cells and induced pluripotent stem cells (iPSCs) are being investigated as sources of retinal pigment epithelium (RPE), photoreceptors, retinal progenitors, and retinal organoids for degenerative retinal diseases. Recent clinical studies have demonstrated encouraging safety profiles for selected RPE-based therapies, while advances in retinal organoids, genome editing, biomaterials, and tissue engineering are opening new possibilities for functional retinal reconstruction. Nevertheless, substantial challenges remain, including cell heterogeneity, incomplete maturation, limited survival and integration, immune rejection, tumorigenic potential, manufacturing variability, and the absence of standardized potency assays. The field is therefore moving from conventional cell transplantation toward integrated regenerative strategies combining precisely characterized cells, engineered microenvironments, genetic correction, biomaterials, and advanced monitoring. This editorial discusses the current state and future direction of ocular stem-cell therapy and emphasizes the need to translate biological promise into reproducible, evidence-based clinical benefit.