Department of Surgery, Medical University of South Carolina, Charleston, SC, 29425, USA.
International Graduate Program Medical Neuroscience, Charité-Universitätsmedizin Berlin, Berlin, Germany.
Stem Cell Res Ther. 2023 Apr 7;14(1):66. doi: 10.1186/s13287-023-03287-7.
Mesenchymal stromal/stem cells (MSCs) are widely utilized in cell therapy because of their robust immunomodulatory and regenerative properties. Their paracrine activity is one of the most important features that contribute to their efficacy. Recently, it has been demonstrated that the production of various factors via extracellular vesicles, especially exosomes, governs the principal efficacy of MSCs after infusion in experimental models. Compared to MSCs themselves, MSC-derived exosomes (MSC-Exos) have provided significant advantages by efficiently decreasing unfavorable adverse effects, such as infusion-related toxicities. MSC-Exos is becoming a promising cell-free therapeutic tool and an increasing number of clinical studies started to assess the therapeutic effect of MSC-Exos in different diseases. In this review, we summarized the ongoing and completed clinical studies using MSC-Exos for immunomodulation, regenerative medicine, gene delivery, and beyond. Additionally, we summarized MSC-Exos production methods utilized in these studies with an emphasis on MSCs source, MSC-Exos isolation methods, characterization, dosage, and route of administration. Lastly, we discussed the current challenges and future directions of exosome utilization in different clinical studies as a novel therapeutic strategy.
间充质基质/干细胞（MSCs）因其强大的免疫调节和再生特性而被广泛应用于细胞治疗。它们的旁分泌活性是其发挥疗效的最重要特征之一。最近，已经证明通过细胞外囊泡，特别是外泌体，产生各种因子，控制了 MSC 在实验模型中输注后的主要疗效。与 MSC 本身相比，MSC 衍生的外泌体（MSC-Exos）通过有效减少输注相关毒性等不利副作用提供了显著优势。MSC-Exos 正成为一种有前途的无细胞治疗工具，越来越多的临床研究开始评估 MSC-Exos 在不同疾病中的治疗效果。在这篇综述中，我们总结了正在进行和已完成的使用 MSC-Exos 进行免疫调节、再生医学、基因传递等的临床研究。此外，我们还总结了这些研究中使用的 MSC-Exos 生产方法，重点介绍了 MSC 来源、MSC-Exos 分离方法、表征、剂量和给药途径。最后，我们讨论了外泌体作为一种新型治疗策略在不同临床研究中的当前挑战和未来方向。
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