Lin Yu, Ren Xiang, Chen Yongjiang, Chen Danian
The Research Laboratory of Ophthalmology and Vision Sciences, State Key Laboratory of Biotherapy, West China Hospital, Sichuan University, Chengdu, China.
The Department of Ophthalmology, West China Hospital, Sichuan University, Chengdu, China.
Front Neurosci. 2021 Jan 21;14:617377. doi: 10.3389/fnins.2020.617377. eCollection 2020.
Retinal degenerative diseases (RDDs) are a group of diseases contributing to irreversible vision loss with yet limited therapies. Stem cell-based therapy is a promising novel therapeutic approach in RDD treatment. Mesenchymal stromal/stem cells (MSCs) have emerged as a leading cell source due to their neurotrophic and immunomodulatory capabilities, limited ethical concerns, and low risk of tumor formation. Several pre-clinical studies have shown that MSCs have the potential to delay retinal degeneration, and recent clinical trials have demonstrated promising safety profiles for the application of MSCs in retinal disease. However, some of the clinical-stage MSC therapies have been unable to meet primary efficacy end points, and severe side effects were reported in some retinal "stem cell" clinics. In this review, we provide an update of the interaction between MSCs and the RDD microenvironment and discuss how to balance the therapeutic potential and safety concerns of MSCs' ocular application.
视网膜退行性疾病(RDDs)是一类导致不可逆视力丧失且治疗方法有限的疾病。基于干细胞的疗法是RDD治疗中一种有前景的新型治疗方法。间充质基质/干细胞(MSCs)因其神经营养和免疫调节能力、伦理问题有限以及肿瘤形成风险低,已成为主要的细胞来源。多项临床前研究表明,MSCs有延缓视网膜变性的潜力,最近的临床试验也证明了MSCs应用于视网膜疾病具有良好的安全性。然而,一些临床阶段的MSC疗法未能达到主要疗效终点,并且在一些视网膜“干细胞”诊所报告了严重的副作用。在本综述中,我们提供了MSCs与RDD微环境之间相互作用的最新情况,并讨论了如何平衡MSCs眼部应用的治疗潜力和安全问题。