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多能干细胞向临床应用推进。

Pluripotent stem cells progressing to the clinic.

机构信息

Monash University and Hudson Institute for Medical Research, 27-31 Right Street, Clayton, Victoria 3168, Australia.

California Institute for Regenerative Medicine (CIRM), 1999 Harrison Street, Suite 1650, Oakland, 94612, USA.

出版信息

Nat Rev Mol Cell Biol. 2016 Mar;17(3):194-200. doi: 10.1038/nrm.2016.10.

Abstract

Basic experimental stem cell research has opened up the possibility of many diverse clinical applications; however, translation to clinical trials has been restricted to only a few diseases. To broaden this clinical scope, pluripotent stem cell derivatives provide a uniquely scalable source of functional differentiated cells that can potentially repair damaged or diseased tissues to treat a wide spectrum of diseases and injuries. However, gathering sound data on their distribution, longevity, function and mechanisms of action in host tissues is imperative to realizing their clinical benefit. The large-scale availability of treatments involving pluripotent stem cells remains some years away, because of the long and demanding regulatory pathway that is needed to ensure their safety.

摘要

基础的实验性干细胞研究已经开启了许多不同临床应用的可能性;然而,将其转化为临床试验的成果仅限于少数几种疾病。为了拓宽这一临床应用范围,多能干细胞衍生细胞提供了一个独特的、可规模化的功能性分化细胞来源,这些细胞有可能修复受损或患病的组织,从而治疗广泛的疾病和损伤。然而,为了实现其临床应用,获得关于它们在宿主组织中的分布、寿命、功能和作用机制的可靠数据是至关重要的。由于需要通过漫长而苛刻的监管途径来确保其安全性,涉及多能干细胞的治疗手段在几年内还无法大规模应用。

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