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用于临床应用和细胞产品的人类诱导多能干细胞。

Human iPS Cells for Clinical Applications and Cellular Products.

机构信息

Centre for iPS Cell Research and Application (CiRA) and Institute for the Advanced Study of Human Biology (ASHBi), Kyoto University, Kyoto, Japan.

出版信息

Handb Exp Pharmacol. 2023;281:3-27. doi: 10.1007/164_2023_643.

Abstract

Human induced pluripotent stem cells (iPSCs), since their discovery in 2007, have rapidly become a starting cell type of choice for the differentiation of many mature cell types. Their flexibility, amenability to gene editing and functional equivalence to embryonic stem cells ensured their subsequent adoption by many manufacturing processes for cellular products. In this chapter, we will discuss the process whereby iPSCs are generated, key quality control steps which should be considered during manufacturing, the application of good manufacturing practice to production processes and iPSC-derived cellular products which are already undergoing clinical trials. iPSCs provide a new avenue for the next generation of cellular therapeutics and by combining new differentiation protocols, quality control and reproducible manufacturing, iPSC-derived cellular products could provide treatments for many currently untreatable diseases, allowing the large-scale manufacture of high-quality cell therapies.

摘要

人诱导多能干细胞(iPSCs)自 2007 年发现以来,迅速成为许多成熟细胞类型分化的首选起始细胞类型。其灵活性、基因编辑的易操作性以及与胚胎干细胞的功能等同性,确保了它们随后被许多细胞产品的制造工艺所采用。在本章中,我们将讨论生成 iPSCs 的过程、制造过程中应考虑的关键质量控制步骤、良好生产规范在生产过程中的应用以及正在进行临床试验的 iPSC 衍生细胞产品。iPSCs 为下一代细胞治疗提供了新途径,通过结合新的分化方案、质量控制和可重复的制造,iPSC 衍生的细胞产品可能为许多目前无法治疗的疾病提供治疗方法,从而能够大规模制造高质量的细胞疗法。

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