干细胞移植治疗视网膜变性疾病临床试验现状及前景

Stem cell transplantation for retinal degeneration: current status and future prospects

随着白内障超声乳化手术、角膜移植等治疗技术的普及,白内障和角膜盲这两类主要的致盲性眼病逐步被攻克,而老年性黄斑变性(age related macular degeneration,AMD)、视网膜色素变性(retinitis pigmentosa,RP)和青光眼等视网膜视神经变性类疾病成为威胁人类视力健康的主要疾病。该类疾病的共同特点是:视网膜中的关键细胞(如感光细胞、视网膜色素上皮细胞和神经节细胞)变性、死亡,患者的视功能逐渐丧失至失明。由于变性和凋亡的神经细胞不能再生,干细胞移植成为目前最具前景的治疗策略。

Age-related macular degeneration( AMD) and retinitis pigmentosa( RP),characterized by dysfunction and loss of retinal pigment epithelium( RPE) and photoreceptors and then retinal degeneration,are major causes of blindness. Currently,there is still no curable treatment available. Recent clinical trials showed that stem cell therapy provided a safe and promising efficiency for these retinal diseases.Transplantation of human embryonic stem cells( h ESCs) and induced pluripotent stem( i PS) cells have obtained safe outcomes for RPE replacement and achieved certain efficacy in treatment of AMD. Fetal retinal progenitor cells for photoreceptor replacement have also confirmed long-term safety and feasibility in vision repair for RP patients. However,there still exist some challenges in this field,including developing novo transplantation technique,ensuring long-term cellular survival and promoting the integration of transplanted cells into the retina. This article provided an analysis of ongoing studies and challenges ahead in this aspect.