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基于干细胞的年龄相关性黄斑变性治疗:现状与前景

Stem cell-based therapies for age-related macular degeneration: current status and prospects.

作者信息

Mu Yalin, Zhao Manli, Su Guangming

机构信息

Department of Ophthalmology, Yellow River Hospital, Henan University of Science and Technology Sanmenxia City, Henan Province, People's Republic of China.

出版信息

Int J Clin Exp Med. 2014 Nov 15;7(11):3843-52. eCollection 2014.

Abstract

Age-related macular degeneration (AMD) is one of the major causes of irreversible blindness both in developed and developing countries. During the past decades, the managements of neovascular AMD (wet AMD) have dramatically progressed. However, still no effective treatment for non-neovascular AMD (dry AMD) which was characterized by geographic macular atrophy. Recent advances in stem cell sciences have demonstrated that retinal pigment epithelium (RPE) cells can be generated from several types of stem cells (including embryonic stem cells, induced pluripotent stem cells, mesenchymal stem cells, et al) by cell co-culturing or defined factors. Additionally, studies also showed that visual function could be recovered by transplantation of these cells into subretinal space in vivo. Moreover, the United States Food and Drug Administration already approved several clinical trials to evaluate the efficiencies of stem cell based cell transplantation for dry AMD patients. Till now, a few patients enrolled in these studies achieved promising outcomes. This review will summarize recent advances in stem cell based RPE differentiation, transplantation, and the preliminary results of clinical trials. The obstacles and prospects in this field will also be discussed.

摘要

年龄相关性黄斑变性(AMD)是发达国家和发展中国家不可逆性失明的主要原因之一。在过去几十年中,新生血管性AMD(湿性AMD)的治疗取得了显著进展。然而,对于以地图样黄斑萎缩为特征的非新生血管性AMD(干性AMD),仍然没有有效的治疗方法。干细胞科学的最新进展表明,通过细胞共培养或特定因子,可以从几种类型的干细胞(包括胚胎干细胞、诱导多能干细胞、间充质干细胞等)生成视网膜色素上皮(RPE)细胞。此外,研究还表明,将这些细胞移植到体内视网膜下间隙可恢复视觉功能。此外,美国食品药品监督管理局已经批准了几项临床试验,以评估基于干细胞的细胞移植对干性AMD患者的疗效。到目前为止,参与这些研究的少数患者取得了令人鼓舞的结果。本综述将总结基于干细胞的RPE分化、移植的最新进展以及临床试验的初步结果。还将讨论该领域的障碍和前景。

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