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基于干细胞/祖细胞的移植治疗视网膜变性:临床试验综述。

Stem/progenitor cell-based transplantation for retinal degeneration: a review of clinical trials.

机构信息

Department of Ophthalmology, Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai Key Laboratory of Orbital Diseases and Ocular Oncology, Shanghai, 200011, P.R. China.

出版信息

Cell Death Dis. 2020 Sep 23;11(9):793. doi: 10.1038/s41419-020-02955-3.

Abstract

Retinal degeneration (RD) is one of the dominant causes of irreversible vision impairment and blindness worldwide. However, the current effective therapeutics for RD in the ophthalmologic clinic are unclear and controversial. In recent years, extensively investigated stem/progenitor cells-including retinal progenitor cells (RPCs), embryonic stem cells (ESCs), induced pluripotent stem cells (iPSCs) and mesenchymal stromal cells (MSCs)-with proliferation and multidirectional differentiation potential have presented opportunities to revolutionise the ultimate clinical management of RD. Herein, we provide a comprehensive overview on the progression of clinical trials for RD treatment using four types of stem/progenitor cell-based transplantation to replace degenerative retinal cells and/or to supplement trophic factors from the aspects of safety, effectiveness and their respective advantages and disadvantages. In addition, we also discuss the emerging role of stem cells in the secretion of multifunctional nanoscale exosomes by which stem cells could be further exploited as a potential RD therapy. This review will facilitate the understanding of scientists and clinicians of the enormous promise of stem/progenitor cell-based transplantation for RD treatment, and provide incentive for superior employment of such strategies that may be suitable for treatment of other diseases, such as stroke and ischaemia-reperfusion injury.

摘要

视网膜变性(RD)是全球范围内导致不可逆转视力损害和失明的主要原因之一。然而,目前眼科临床治疗 RD 的有效疗法尚不清楚,存在争议。近年来,具有增殖和多向分化潜能的广泛研究的干细胞/祖细胞——包括视网膜祖细胞(RPCs)、胚胎干细胞(ESCs)、诱导多能干细胞(iPSCs)和间充质基质细胞(MSCs)——为最终临床管理 RD 带来了变革的机会。在此,我们从安全性、有效性及其各自的优缺点等方面,对四种基于干细胞/祖细胞的移植治疗 RD 的临床试验进展进行了全面综述,以替代变性视网膜细胞和/或补充神经营养因子。此外,我们还讨论了干细胞在分泌多功能纳米级外泌体中的新作用,这使得干细胞可以进一步被开发为 RD 的潜在治疗方法。本综述将有助于科学家和临床医生了解基于干细胞/祖细胞的移植治疗 RD 的巨大潜力,并为更好地利用这些策略提供动力,这些策略可能适用于治疗其他疾病,如中风和缺血再灌注损伤。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b454/7511341/f56126a843e4/41419_2020_2955_Fig1_HTML.jpg

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