Pluripotent stem cell therapy for retinal diseases.
Pluripotent stem cell therapy for retinal diseases.
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DOI：
10.21037/atm-20-4747
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发表时间：
2021-08
影响因子：
--
通讯作者：
Singh MS
中科院分区：
文献类型：
--
作者：
Ahmed I;Johnston RJ Jr;Singh MS
多能干细胞（PSC），包括人胚胎干细胞（hESC）和诱导多能干细胞（iPSC），已被用于研究疾病过程的发展，并作为多器官系统中的潜在疗法。近年来，人们越来越关注使用基于PSC的移植来治疗视网膜疾病，其中视网膜细胞已经通过变性而功能受损或丧失。由神经元组织组成的视网膜提供了一个很好的系统来测试基于PSC的移植的治疗效用，因为它的可访问性和高分辨率成像技术的可用性来评估效果。在视网膜疾病的动物模型中的临床前试验已经显示，在PSC衍生的光感受器或视网膜色素上皮（RPE）细胞的视网膜下移植后，视觉结果得到改善。本文综述了PSC在视网膜疾病治疗中的临床前研究和临床试验。迄今为止，在年龄相关性黄斑变性（AMD）和Stargardt病（STGD 1）患者中进行的几项I/II期临床试验已经证明了PSC衍生的RPE移植的安全性和可行性。使用PSC衍生的RPE或感光细胞治疗AMD、STGD 1以及视网膜色素变性（RP）的其他I/II期临床试验目前正在进行中。随着这一领域的不断发展，其他技术可以通过自体细胞的基因编辑，移植更复杂的细胞结构（如类器官）以及通过新型成像技术监测移植细胞来增强PSC衍生细胞移植。
Pluripotent stem cells (PSCs), which include human embryonic stem cells (hESCs) and induced pluripotent stem cell (iPSC), have been used to study development of disease processes, and as potential therapies in multiple organ systems. In recent years, there has been increasing interest in the use of PSC-based transplantation to treat disorders of the retina in which retinal cells have been functionally damaged or lost through degeneration. The retina, which consists of neuronal tissue, provides an excellent system to test the therapeutic utility of PSC-based transplantation due to its accessibility and the availability of high-resolution imaging technology to evaluate effects. Preclinical trials in animal models of retinal diseases have shown improvement in visual outcomes following subretinal transplantation of PSC-derived photoreceptors or retinal pigment epithelium (RPE) cells. This review focuses on preclinical studies and clinical trials exploring the use of PSCs for retinal diseases. To date, several phase I/II clinical trials in patients with age-related macular degeneration (AMD) and Stargardt disease (STGD1) have demonstrated the safety and feasibility of PSC-derived RPE transplantation. Additional phase I/II clinical trials using PSC-derived RPE or photoreceptor cells for the treatment of AMD, STGD1, and also retinitis pigmentosa (RP) are currently in the pipeline. As this field continues to evolve, additional technologies may enhance PSC-derived cell transplantation through gene-editing of autologous cells, transplantation of more complex cellular structures such as organoids, and monitoring of transplanted cells through novel imaging technologies.
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