Xie Nina, Tang Beisha
Department of Neurology, Xiangya Hospital, Central South University, Changsha, Hunan 410008, China.
Stem Cells Int. 2016;2016:6484713. doi: 10.1155/2016/6484713. Epub 2016 Jan 6.
In principle, induced pluripotent stem cells (iPSCs) are generated from somatic cells by reprogramming and gaining the capacity to self-renew indefinitely as well as the ability to differentiate into cells of different lineages. Human iPSCs have absolute advantages over human embryonic stem cells (ESCs) and animal models in disease modeling, drug screening, and cell replacement therapy. Since Takahashi and Yamanaka first described in 2007 that iPSCs can be generated from human adult somatic cells by retroviral transduction of the four transcription factors, Oct3/4, Sox2, Klf4, and c-Myc, disease specific iPSC lines have sprung up worldwide like bamboo shoots after a spring rain, making iPSC one of the hottest and fastest moving topics in modern science. The craze for iPSCs has spread throughout main branches of clinical medicine, covering neurology, hematology, cardiology, endocrinology, hepatology, ophthalmology, and so on. Here in this paper, we will focus on the clinical application of human iPSCs in disease modeling, drug screening, and cell replacement therapy for neurological diseases.
原则上，诱导多能干细胞（iPSC）是通过重编程由体细胞产生的，具有无限自我更新的能力以及分化为不同谱系细胞的能力。在疾病建模、药物筛选和细胞替代治疗方面，人类iPSC相对于人类胚胎干细胞（ESC）和动物模型具有绝对优势。自2007年高桥和山中首次描述通过逆转录病毒转导四个转录因子Oct3/4、Sox2、Klf4和c-Myc可从人类成体细胞生成iPSC以来，疾病特异性iPSC系如雨后春笋般在全球涌现，使iPSC成为现代科学中最热门、发展最快的话题之一。对iPSC的狂热已蔓延到临床医学的各个主要分支，涵盖神经学、血液学、心脏病学、内分泌学、肝脏病学、眼科学等。在本文中，我们将重点关注人类iPSC在神经疾病的疾病建模、药物筛选和细胞替代治疗中的临床应用。
