Tsujimoto Hiraku, Osafune Kenji
Center for iPS Cell Research and Application (CiRA), Kyoto University, Japan.
RegeNephro Co., Ltd., MIC bldg. Graduate School of Medicine, Kyoto University, Japan.
FEBS J. 2022 Dec;289(23):7274-7291. doi: 10.1111/febs.16162. Epub 2021 Aug 27.
Regenerative medicine using iPS cell technologies has progressed remarkably in recent years. In this review, we summarize these technologies and their clinical application. First, we discuss progress in the establishment of iPS cells, including the HLA-homo iPS cell stock project in Japan and the advancement of low antigenic iPS cells using genome-editing technology. Then, we describe iPS cell-based therapies in or approaching clinical application, including those for ophthalmological, neurological, cardiac, hematological, cartilage, and metabolic diseases. Next, we introduce disease models generated from patient iPS cells and successfully used to identify therapeutic agents for intractable diseases. Clinical medicine using iPS cells has advanced safely and effectively by making full use of current scientific standards, but tests on cell safety need to be further developed and validated. The next decades will see the further spread of iPS cell technology-based regenerative medicine.
近年来，利用诱导多能干细胞（iPS细胞）技术的再生医学取得了显著进展。在本综述中，我们总结了这些技术及其临床应用。首先，我们讨论了iPS细胞建立方面的进展，包括日本的人类白细胞抗原（HLA）同型iPS细胞库项目，以及利用基因组编辑技术推进低抗原性iPS细胞的研究。然后，我们描述了已进入或即将进入临床应用的基于iPS细胞的疗法，包括用于眼科、神经科、心脏科、血液科、软骨和代谢疾病的疗法。接下来，我们介绍了由患者iPS细胞生成并成功用于鉴定难治性疾病治疗药物的疾病模型。通过充分利用当前的科学标准，基于iPS细胞的临床医学已安全有效地取得进展，但细胞安全性测试仍需进一步发展和验证。未来几十年，基于iPS细胞技术的再生医学将得到进一步推广。
