Tang Zhimin, Zhang Yi, Wang Yuyao, Zhang Dandan, Shen Bingqiao, Luo Min, Gu Ping
Department of Ophthalmology, Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, 200011, People's Republic of China.
J Transl Med. 2017 May 10;15(1):99. doi: 10.1186/s12967-017-1183-y.
Retinal degeneration (RD), such as age-related macular degeneration (AMD) and retinitis pigmentosa, is one of the leading causes of blindness. Presently, no satisfactory therapeutic options are available for these diseases principally because the retina and retinal pigmented epithelium (RPE) do not regenerate, although wet AMD can be prevented from further progression by anti-vascular endothelial growth factor therapy. Nevertheless, stem/progenitor cell approaches exhibit enormous potential for RD treatment using strategies mainly aimed at the rescue and replacement of photoreceptors and RPE. The sources of stem/progenitor cells are classified into two broad categories in this review, which are (1) ocular-derived progenitor cells, such as retinal progenitor cells, and (2) non-ocular-derived stem cells, including embryonic stem cells, induced pluripotent stem cells, and mesenchymal stromal cells. Here, we discuss in detail the progress in the study of four predominant stem/progenitor cell types used in animal models of RD. A short overview of clinical trials involving the stem/progenitor cells is also presented. Currently, stem/progenitor cell therapies for RD still have some drawbacks such as inhibited proliferation and/or differentiation in vitro (with the exception of the RPE) and limited long-term survival and function of grafts in vivo. Despite these challenges, stem/progenitor cells represent the most promising strategy for RD treatment in the near future.
视网膜变性（RD），如年龄相关性黄斑变性（AMD）和视网膜色素变性，是导致失明的主要原因之一。目前，这些疾病尚无令人满意的治疗选择，主要是因为视网膜和视网膜色素上皮（RPE）无法再生，尽管抗血管内皮生长因子疗法可防止湿性AMD进一步发展。然而，干细胞/祖细胞方法在RD治疗中显示出巨大潜力，其策略主要旨在挽救和替换光感受器及RPE。在本综述中，干细胞/祖细胞的来源分为两大类，即（1）眼源性祖细胞，如视网膜祖细胞，以及（2）非眼源性干细胞，包括胚胎干细胞、诱导多能干细胞和间充质基质细胞。在此，我们详细讨论了在RD动物模型中使用的四种主要干细胞/祖细胞类型的研究进展。还简要概述了涉及干细胞/祖细胞的临床试验。目前，用于RD的干细胞/祖细胞疗法仍存在一些缺点，如体外增殖和/或分化受到抑制（RPE除外），以及移植物在体内的长期存活和功能有限。尽管存在这些挑战，但干细胞/祖细胞仍是近期RD治疗最有前景的策略。
