Shi Yanhong, Inoue Haruhisa, Wu Joseph C, Yamanaka Shinya
Division of Stem Cell Biology Research, Department of Developmental and Stem Cell Biology, Beckman Research Institute of City of Hope, 1500 East Duarte Road, Duarte, California 91010, USA.
Center for iPS Cell Research and Application (CiRA), Kyoto University, 53 Kawahara-cho Shogoin, Sakyo-ku, Kyoto 606-8507, Japan.
Nat Rev Drug Discov. 2017 Feb;16(2):115-130. doi: 10.1038/nrd.2016.245. Epub 2016 Dec 16.
Since the advent of induced pluripotent stem cell (iPSC) technology a decade ago, enormous progress has been made in stem cell biology and regenerative medicine. Human iPSCs have been widely used for disease modelling, drug discovery and cell therapy development. Novel pathological mechanisms have been elucidated, new drugs originating from iPSC screens are in the pipeline and the first clinical trial using human iPSC-derived products has been initiated. In particular, the combination of human iPSC technology with recent developments in gene editing and 3D organoids makes iPSC-based platforms even more powerful in each area of their application, including precision medicine. In this Review, we discuss the progress in applications of iPSC technology that are particularly relevant to drug discovery and regenerative medicine, and consider the remaining challenges and the emerging opportunities in the field.
自十年前诱导多能干细胞（iPSC）技术问世以来，干细胞生物学和再生医学领域取得了巨大进展。人类iPSC已广泛应用于疾病建模、药物发现和细胞治疗开发。新的病理机制已被阐明，源自iPSC筛选的新药正在研发中，并且首个使用人类iPSC衍生产品的临床试验已经启动。特别是，人类iPSC技术与基因编辑和3D类器官的最新进展相结合，使得基于iPSC的平台在其应用的各个领域（包括精准医学）中更加强大。在本综述中，我们讨论了iPSC技术在与药物发现和再生医学特别相关的应用方面取得的进展，并考虑了该领域尚存的挑战和新出现的机遇。
