Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.
JRG Translational Hematology of Congenital Diseases, REBIRTH Cluster of Excellence, Hannover Medical School, Hannover, Germany.
Stem Cells Transl Med. 2019 Apr;8(4):332-339. doi: 10.1002/sctm.18-0134. Epub 2018 Dec 26.
Since their discovery in 2006, induced pluripotent stem cells (iPSCs) have opened up a world of possibilities for regenerative medicine and novel cell-based therapeutics. Now, over a decade later, robust reprogramming and expansion and differentiation protocols have been developed, and iPSC-derived cells have been used in a wide variety of small and large animal models to treat many different diseases. Furthermore, the first iPSC derivatives are on their way into clinical trials. In this line, (i) GMP-compliant generation, cultivation, and differentiation, (ii) preclinical efficacy and safety, as well as (iii) ethical and regulatory compliance of stem cell research represent important aspects that need to be evaluated for proper clinical translation of iPSCs and their derivatives. In this review article, we provide an overview of the current advances and challenges of the clinical translation of iPSC-derived blood cells and highlight the most pressing problems that have to be overcome in the next years. Stem Cells Translational Medicine 2019;8:332-339.
自 2006 年发现以来，诱导多能干细胞（iPSCs）为再生医学和新型基于细胞的治疗开辟了无限可能。现在，十多年过去了，已经开发出了强大的重编程和扩增分化方案，并且 iPSC 衍生细胞已被用于各种小型和大型动物模型中，以治疗许多不同的疾病。此外，首批 iPSC 衍生物即将进入临床试验。在此过程中，（i）符合 GMP 的生成、培养和分化，（ii）临床前疗效和安全性，以及（iii）干细胞研究的伦理和监管合规性是需要进行评估的重要方面，以实现 iPSC 及其衍生物的适当临床转化。在这篇综述文章中，我们概述了 iPSC 衍生血细胞临床转化的最新进展和挑战，并强调了未来几年需要克服的最紧迫问题。《Stem Cells Translational Medicine》2019 年；8:332-339.
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