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诱导多能干细胞,人类治疗应用的巨大飞跃。

Induced pluripotent stem cells, a giant leap for mankind therapeutic applications.

作者信息

Bragança José, Lopes João André, Mendes-Silva Leonardo, Almeida Santos João Miguel

机构信息

Department of Biomedical Sciences and Medicine, University of Algarve, Faro 8005-139, Portugal.

出版信息

World J Stem Cells. 2019 Jul 26;11(7):421-430. doi: 10.4252/wjsc.v11.i7.421.

DOI:10.4252/wjsc.v11.i7.421
PMID:31396369
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC6682501/
Abstract

Induced pluripotent stem cells (iPSC) technology has propelled the field of stem cells biology, providing new cells to explore the molecular mechanisms of pluripotency, cancer biology and aging. A major advantage of human iPSC, compared to the pluripotent embryonic stem cells, is that they can be generated from virtually any embryonic or adult somatic cell type without destruction of human blastocysts. In addition, iPSC can be generated from somatic cells harvested from normal individuals or patients, and used as a cellular tool to unravel mechanisms of human development and to model diseases in a manner not possible before. Besides these fundamental aspects of human biology and physiology that are revealed using iPSC or iPSC-derived cells, these cells hold an immense potential for cell-based therapies, and for the discovery of new or personalized pharmacological treatments for many disorders. Here, we review some of the current challenges and concerns about iPSC technology. We introduce the potential held by iPSC for research and development of novel health-related applications. We briefly present the efforts made by the scientific and clinical communities to create the necessary guidelines and regulations to achieve the highest quality standards in the procedures for iPSC generation, characterization and long-term preservation. Finally, we present some of the audacious and pioneer clinical trials in progress with iPSC-derived cells.

摘要

诱导多能干细胞(iPSC)技术推动了干细胞生物学领域的发展,为探索多能性、癌症生物学和衰老的分子机制提供了新的细胞。与多能胚胎干细胞相比,人类iPSC的一个主要优势在于,它们几乎可以从任何胚胎或成体体细胞类型中产生,而无需破坏人类囊胚。此外,iPSC可以从正常个体或患者采集的体细胞中产生,并用作一种细胞工具,以前所未有的方式揭示人类发育机制并模拟疾病。除了使用iPSC或iPSC衍生细胞所揭示的人类生物学和生理学的这些基本方面外,这些细胞在基于细胞的治疗以及发现针对许多疾病的新的或个性化药物治疗方面具有巨大潜力。在此,我们综述了当前关于iPSC技术的一些挑战和问题。我们介绍了iPSC在新型健康相关应用研发方面的潜力。我们简要介绍了科学界和临床界为制定必要的指导方针和法规所做的努力,以在iPSC生成、表征和长期保存程序中实现最高质量标准。最后,我们介绍了一些正在进行的使用iPSC衍生细胞的大胆且开创性的临床试验。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/548a/6682501/359e34705466/WJSC-11-421-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/548a/6682501/359e34705466/WJSC-11-421-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/548a/6682501/359e34705466/WJSC-11-421-g001.jpg

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Partial reprogramming induces a steady decline in epigenetic age before loss of somatic identity.部分重编程会导致表观遗传年龄在丧失体身份之前稳步下降。
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Challenges in designing and executing clinical trials in a dish studies.在培养皿研究中设计和开展临床试验所面临的挑战。
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