Karagiannis Peter, Nakauchi Ayaka, Yamanaka Shinya
Center for iPS Cell Research and Application, Kyoto University, Kyoto, Japan.
JMA J. 2018 Sep 28;1(1):6-14. doi: 10.31662/jmaj.2018-0005.
Induced pluripotent stem cells (iPSCs) describe somatic cells that have been reprogrammed to the pluripotent state. From a scientific perspective, their discovery has provided a molecular roadmap for turning on and off cell identities, effectively allowing any cell type to have its identity changed into any other cell type. They also act as a human model for understanding the development of every cell and organ in the body. In addition, because they can be prepared from patients, iPSCs offer a unique human model for studying disease development, including many diseases that are generally diagnosed at a late stage of their development. These models have provided new insights on the pathogenesis and new targets to prevent or reverse the disease development process. Indeed, clinical studies on compounds based on drug screening hits in human iPSC disease models have begun. Because of their proliferation and differentiation capacity, iPSCs can also be used to prepare cells for transplantations, and related clinical studies using iPSC-based cell therapies are ongoing. The combination of iPSCs with other technologies or therapeutic strategies is expected to expand their medical benefits. In this review, we consider medical accomplishments based on iPSC research and future ones that can be anticipated.
诱导多能干细胞（iPSC）是指已被重编程为多能状态的体细胞。从科学角度来看，它们的发现为开启和关闭细胞身份提供了分子路线图，从而有效地使任何细胞类型的身份转变为任何其他细胞类型。它们还可作为一种人类模型，用于理解体内每个细胞和器官的发育过程。此外，由于iPSC可以从患者身上制备，因此为研究疾病发展提供了独特的人类模型，包括许多通常在疾病发展后期才被诊断出来的疾病。这些模型为疾病的发病机制提供了新的见解，并为预防或逆转疾病发展过程提供了新的靶点。事实上，基于人类iPSC疾病模型中药物筛选结果的化合物的临床研究已经开始。由于iPSC具有增殖和分化能力，它们还可用于制备移植用细胞，基于iPSC的细胞疗法的相关临床研究正在进行中。iPSC与其他技术或治疗策略的结合有望扩大其医疗益处。在这篇综述中，我们将探讨基于iPSC研究的医学成就以及未来可预期的成就。
