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多能干细胞的治疗应用:挑战与风险

Therapeutic Application of Pluripotent Stem Cells: Challenges and Risks.

作者信息

Martin Ulrich

机构信息

Leibniz Research Laboratories for Biotechnology and Artificial Organs (LEBAO), Department of Cardiothoracic, Transplantation and Vascular Surgery, REBIRTH Cluster of Excellence, German Center for Lung Research, Hannover Medical School, Hannover, Germany.

出版信息

Front Med (Lausanne). 2017 Dec 14;4:229. doi: 10.3389/fmed.2017.00229. eCollection 2017.

Abstract

Stem-cell-based therapies are considered to be promising and innovative but complex approaches. Induced pluripotent stem cells (iPSCs) combine the advantages of adult stem cells with the hitherto unique characteristics of embryonic stem cells (ESCs). Major progress has already been achieved with regard to reprogramming technology, but also regarding targeted genome editing and scalable expansion and differentiation of iPSCs and ESCs, in some cases yielding highly enriched preparations of well-defined cell lineages at clinically required dimensions. It is noteworthy, however, that for many applications critical requirements such as the targeted specification into distinct cellular subpopulations and a proper cell maturation remain to be achieved. Moreover, current hurdles such as low survival rates and insufficient functional integration of cellular transplants remain to be overcome. Nevertheless, PSC technologies obviously have come of age and matured to a stage where various clinical applications of PSC-based cellular therapies have been initiated and are conducted.

摘要

基于干细胞的疗法被认为是有前景、创新但复杂的方法。诱导多能干细胞(iPSC)结合了成体干细胞的优势以及胚胎干细胞(ESC)迄今独特的特性。在重编程技术方面已经取得了重大进展,在诱导多能干细胞和胚胎干细胞的靶向基因组编辑、可扩展扩增及分化方面也取得了进展,在某些情况下能够获得临床所需规模的高度富集的特定细胞谱系制剂。然而,值得注意的是,对于许多应用而言,将细胞靶向分化为不同细胞亚群以及实现适当的细胞成熟等关键要求仍有待实现。此外,诸如细胞移植存活率低和功能整合不足等当前障碍仍有待克服。尽管如此,多能干细胞技术显然已经成熟,发展到了开展各种基于多能干细胞的细胞疗法临床应用的阶段。

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