Transplantation of induced pluripotent stem cell (iPSC)-derived retinal organoids into retinal disease animal models has yielded promising results, and several clinical trials on iPSC-derived retinal pigment epithelial cell transplantation have confirmed its safety. In this study, we performed allogeneic iPSC-derived retinal organoid sheet transplantation in two subjects with advanced retinitis pigmentosa (jRCTa050200027). The primary endpoint was the survival and safety of the transplanted retinal organoid sheets in the first year post-transplantation. The secondary endpoints were the safety of the transplantation procedure and visual function evaluation. The grafts survived in a stable condition for 2 years, and the retinal thickness increased at the transplant site without serious adverse events in both subjects. Changes in visual function were less progressive than those of the untreated eye during the follow-up. Allogeneic iPSC-derived retinal organoid sheet transplantation is a potential therapeutic approach, and the treatment's safety and efficacy for visual function should be investigated further.

将诱导多能干细胞（iPSC）来源的视网膜类器官移植到视网膜疾病动物模型中已取得了可喜的结果，并且多项关于iPSC来源的视网膜色素上皮细胞移植的临床试验已证实其安全性。在这项研究中，我们对两名患有晚期色素性视网膜炎的受试者进行了同种异体 iPSC 衍生的视网膜类器官片移植(jRCTa050200027)。主要终点是移植后第一年移植的视网膜类器官片的存活率和安全性。次要终点是移植手术的安全性和视功能评估。两名受试者的移植物在稳定状态下存活了 2 年，移植部位的视网膜厚度有所增加，没有发生严重的不良事件。在随访期间，与未治疗的眼睛相比，视功能的变化进展缓慢。同种异体 iPSC 来源的视网膜类器官片移植是一种潜在的治疗方法，该治疗的安全性和视功能的有效性应进一步研究。