关键词: 致盲性眼病, 干细胞治疗, 基因治疗, 再生医学, 临床转化

Abstract: Ophthalmic regenerative medicine is critically reliant on advancements in stem cell and gene therapy technologies. Following the issuance of China's first production license for a stem cell drug, this field has transitioned into a new phase of industrialization. This paper analyzes the pathological features of four major blinding eye diseases (corneal, lens, glaucoma, and retinal/optic nerve diseases), the limitations of current clinical treatment protocols, and the research progress of stem cell and gene therapies. It highlights that future technical challenges to address include improving the survival rate of transplanted cells and overcoming the capacity limitations of gene vectors. By 2030, it is anticipated that the treatment paradigm will evolve from "delaying blindness" to "visual enhancement", ultimately achieving the goal of "what you see is what you get". (Ophthalmol CHN, 2025, 34: 169-179)

Key words:  blinding eye diseases, stem cell therapy, gene therapy, regenerative medicine, clinical translation