This single-arm, open-label, first-in-human interventional study in Japan aimed to assess the safety and feasibility of transplanting induced pluripotent stem-cell-derived corneal epithelium in patients with limbal stem cell deficiency. The objective was to explore a regenerative approach to corneal repair.
Methods and Findings:
Patients with unilateral limbal stem cell deficiency received transplants of corneal epithelial sheets derived from induced pluripotent stem cells. The primary outcomes measured were safety and epithelialization of the corneal surface. The study reported successful engraftment with no serious adverse events, and improvement in corneal surface stability was observed.
Conclusion:
The transplantation of induced pluripotent stem-cell-derived corneal epithelium appears to be a safe and feasible treatment for limbal stem cell deficiency. Further studies with larger cohorts and longer follow-up are necessary to confirm efficacy and long-term safety.
