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多能干细胞疗法的临床转化:挑战与考量。

Clinical Translation of Pluripotent Stem Cell Therapies: Challenges and Considerations.

机构信息

Université de Paris, PARCC, INSERM, 75015 Paris, France.

Université de Paris, PARCC, INSERM, 75015 Paris, France; Department of Cardiovascular Surgery, Hôpital Européen Georges Pompidou 20, rue Leblanc, 75015 Paris, France.

出版信息

Cell Stem Cell. 2019 Nov 7;25(5):594-606. doi: 10.1016/j.stem.2019.10.001.

DOI:10.1016/j.stem.2019.10.001
PMID:31703770
Abstract

Although the clinical outcomes of cell therapy trials have not met initial expectations, emerging evidence suggests that injury-mediated tissue damage might benefit from the delivery of cells or their secreted products. Pluripotent stem cells (PSCs) are promising cell sources primarily because of their capacity to generate stage- and lineage-specific differentiated derivatives. However, they carry inherent challenges for safe and efficacious clinical translation. This Review describes completed or ongoing trials of PSCs, discusses their potential mechanisms of action, and considers how to address the challenges required for them to become a major therapy, using heart repair as a case study.

摘要

尽管细胞治疗试验的临床结果尚未达到最初的预期,但新出现的证据表明,损伤介导的组织损伤可能受益于细胞或其分泌产物的递送。多能干细胞(PSCs)是很有前途的细胞来源,主要是因为它们具有生成阶段和谱系特异性分化衍生物的能力。然而,它们在安全和有效的临床转化方面存在固有挑战。本综述描述了 PSCs 的已完成或正在进行的试验,讨论了它们的潜在作用机制,并考虑了如何解决使其成为主要治疗方法所需的挑战,以心脏修复为例。

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