角膜内皮功能障碍是角膜失明的主要原因之一，目前传统的治疗选择是使用尸体供体角膜进行角膜移植。然而，在全球范围内，适合的供者移植材料短缺，因此需要探索新的治疗方法。基于诱导多能干细胞（iPSCs）的干细胞再生医学方法提供了一种有希望的解决方案，因为它们具有自我更新的能力，可以从成体体细胞中分化，且可以分化为包括角膜内皮细胞（CECs）在内的各种细胞类型。本综述讨论了发展将iPSCs诱导为CECs的方案的进展和挑战，重点关注不同的培养基配方用于将iPSCs分化为神经嵴细胞（NCCs），并进一步分化为CECs，以及定义iPSCs源CECs的表征方法和标记物。还讨论了iPSC源细胞治疗在临床应用中的难题和解决方案，包括制定符合良好制造规范（GMP）指南的方案。评估了长期体外培养引起的iPSC源CECs的潜在遗传突变风险，以及移植后潜在致瘤性的危险。总之，本综述提供了使用iPSCs治疗角膜内皮功能障碍的进展和障碍的见解。

Corneal endothelial dysfunction is one of the leading causes of corneal blindness, and the current conventional treatment option is corneal transplantation using a cadaveric donor cornea. However, there is a global shortage of suitable donor graft material, necessitating the exploration of novel therapeutic approaches. A stem cell-based regenerative medicine approach using induced pluripotent stem cells (iPSCs) offers a promising solution, as they possess self-renewal capabilities, can be derived from adult somatic cells, and can be differentiated into all cell types including corneal endothelial cells (CECs). This review discusses the progress and challenges in developing protocols to induce iPSCs into CECs, focusing on the different media formulations used to differentiate iPSCs to neural crest cells (NCCs) and subsequently to CECs, as well as the characterization methods and markers that define iPSC-derived CECs. The hurdles and solutions for the clinical application of iPSC-derived cell therapy are also addressed, including the establishment of protocols that adhere to good manufacturing practice (GMP) guidelines. The potential risks of genetic mutations in iPSC-derived CECs associated with long-term in vitro culture and the danger of potential tumorigenicity following transplantation are evaluated. In all, this review provides insights into the advancement and obstacles of using iPSC in the treatment of corneal endothelial dysfunction.