Mesenchymal Stem Cell-Based Therapy as a New Approach for the Treatment of Systemic Sclerosis.

IF 8.4 2区 医学 Q1 ALLERGY
Xiufen Zhuang, Xiao Hu, Shuren Zhang, Xingmin Li, Xiaoying Yuan, Yanhong Wu
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引用次数: 13

Abstract

Systemic sclerosis (SSc) is an intractable autoimmune disease with unmet medical needs. Conventional immunosuppressive therapies have modest efficacy and obvious side effects. Targeted therapies with small molecules and antibodies remain under investigation in small pilot studies. The major breakthrough was the development of autologous haematopoietic stem cell transplantation (AHSCT) to treat refractory SSc with rapidly progressive internal organ involvement. However, AHSCT is contraindicated in patients with advanced visceral involvement. Mesenchymal stem cells (MSCs) which are characterized by immunosuppressive, antifibrotic and proangiogenic capabilities may be a promising alternative option for the treatment of SSc. Multiple preclinical and clinical studies on the use of MSCs to treat SSc are underway. However, there are several unresolved limitations and safety concerns of MSC transplantation, such as immune rejections and risks of tumour formation, respectively. Since the major therapeutic potential of MSCs has been ascribed to their paracrine signalling, the use of MSC-derived extracellular vesicles (EVs)/secretomes/exosomes as a "cell-free" therapy might be an alternative option to circumvent the limitations of MSC-based therapies. In the present review, we overview the current knowledge regarding the therapeutic efficacy of MSCs in SSc, focusing on progresses reported in preclinical and clinical studies using MSCs, as well as challenges and future directions of MSC transplantation as a treatment option for patients with SSc.

间充质干细胞为基础的治疗系统性硬化症的新途径。
系统性硬化症(SSc)是一种难以满足医疗需求的自身免疫性疾病。常规免疫抑制疗法疗效一般,副作用明显。小分子和抗体靶向治疗仍在小规模试点研究中进行研究。主要的突破是自体造血干细胞移植(AHSCT)的发展,以治疗难治性SSc并迅速进展的内部器官受累。然而,AHSCT是晚期内脏受累患者的禁忌症。间充质干细胞(MSCs)具有免疫抑制、抗纤维化和促血管生成能力,可能是治疗SSc的一个有希望的替代选择。使用MSCs治疗SSc的多项临床前和临床研究正在进行中。然而,MSC移植存在一些未解决的限制和安全性问题,例如免疫排斥和肿瘤形成的风险。由于间充质干细胞的主要治疗潜力归因于其旁分泌信号,因此使用间充质干细胞衍生的细胞外囊泡(ev)/分泌组/外泌体作为“无细胞”治疗可能是规避基于间充质干细胞的治疗局限性的另一种选择。在本综述中,我们概述了目前关于MSCs在SSc中的治疗效果的知识,重点介绍了MSCs在临床前和临床研究中的进展,以及MSC移植作为SSc患者治疗选择的挑战和未来方向。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
CiteScore
22.30
自引率
1.10%
发文量
58
审稿时长
6-12 weeks
期刊介绍: Clinical Reviews in Allergy & Immunology is a scholarly journal that focuses on the advancement of clinical management in allergic and immunologic diseases. The journal publishes both scholarly reviews and experimental papers that address the current state of managing these diseases, placing new data into perspective. Each issue of the journal is dedicated to a specific theme of critical importance to allergists and immunologists, aiming to provide a comprehensive understanding of the subject matter for a wide readership. The journal is particularly helpful in explaining how novel data impacts clinical management, along with advancements such as standardized protocols for allergy skin testing and challenge procedures, as well as improved understanding of cell biology. Ultimately, the journal aims to contribute to the improvement of care and management for patients with immune-mediated diseases.
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