干细胞移植疗法治疗视网膜退行性疾病。

Stem Cell Transplantation Therapy for Retinal Degenerative Diseases.

机构信息

The Institute for Translational Medicine, Shandong University/Affiliated Second Hospital, Jinan, Shandong, China.

The Institute for Tissue Engineering and Regenerative Medicine, Liaocheng University/Liaocheng People's Hospital, Liaocheng, Shandong, China.

出版信息

Adv Exp Med Biol. 2020;1266:127-139. doi: 10.1007/978-981-15-4370-8_9.

DOI:10.1007/978-981-15-4370-8_9

PMID:33105499

Abstract

In the past decade, progress in the research on human embryonic stem cells (hESCs) and induced pluripotent stem cells (iPSCs) has provided the solid basis to derive retinal pigment epithelium, photoreceptors, and ganglion cells from hESCs/iPSCs for transplantation therapy of retinal degenerative diseases (RDD). Recently, the iPSC-derived retinal pigment epithelium cells have achieved efficacy in treating patients with age-related macular degeneration (AMD). However, there is still much work to be done about the differentiation of hESCs/iPSCs into clinically required retinal cells and improvement in the methods to deliver the cells into the retina of patients. Here we will review the research advances in stem cell transplantation in animal studies and clinical trials as well as propose the challenges for improving the clinical efficacy and safety of hESCs/iPSCs-derived retinal neural cells in treating retinal degenerative diseases.

摘要

在过去的十年中，人类胚胎干细胞（hESCs）和诱导多能干细胞（iPSCs）研究的进展为从 hESCs/iPSCs 中衍生出视网膜色素上皮细胞、光感受器和神经节细胞提供了坚实的基础，从而用于治疗视网膜退行性疾病（RDD）的移植治疗。最近，iPSC 衍生的视网膜色素上皮细胞已在治疗年龄相关性黄斑变性（AMD）患者方面取得了疗效。然而，hESCs/iPSCs 分化为临床所需的视网膜细胞以及改进将细胞递送至患者视网膜的方法方面仍有许多工作要做。在这里，我们将回顾干细胞移植在动物研究和临床试验中的研究进展，并提出提高 hESCs/iPSCs 衍生的视网膜神经细胞治疗视网膜退行性疾病的临床疗效和安全性的挑战。