摘要
诱导多能干细胞(i PS细胞)在小鼠和人上的成功获取,使干细胞领域的研究进入了一个崭新的时代。干细胞研究是再生医学的重要组成部分,研究干细胞的最终目的是应用干细胞治疗疾病,其在疾病模型建立、药物筛选、细胞移植等方面具有极大的应用潜力。i PSCs是由体细胞诱导分化而成的"多能细胞",具备和胚胎干细胞类似的功能,既解决了ESCs的伦理障碍,又为ESCs的获得提供了一条全新的途径,具有重要的理论和应用价值。i PS细胞不仅打破了道德理论的束缚,而且在再生医学、组织工程和药物发现及评价等方面具有积极的价值。神经系统遗传性疾病发病率居各系统遗传病之首,但其发病的分子机制仍不完全清楚,运用体细胞重编程技术建立的疾病特异性诱导多能干细胞模型将有助于揭示神经系统遗传性疾病的发病机理。近几年i PS细胞最新研究成果表明,利用疾病患者i PS细胞模型已逐渐应用于帕金森氏病、老年性痴呆症、脊髓侧索硬化症、脊髓肌肉萎缩症及舞蹈症等5种常见神经性退行性疾病发病机理的研究。本文主要对i PSc的发展历程,避免病毒基因干扰诱导i PS细胞进行的优化,以及干细胞尤其是i PS细胞移植治疗帕金森病等神经系统疾病的现状及应用前景进行系统阐述与论证。
Induced pluripotent stem cells （iPS cells） got from mice and human were successful, which made the research of stem cell field into a new era. Stem cell research is an important part of regenerative medicine, and the ultimate goal of stem cell research is the application of stem cells for treatment of disease. Furthermore, the research of stem cells has great application potential on the disease models, drug screening, and cell transplantation. As embryonic stem cells, induced pluripotent stem cells are able to self-renew indefinitely and differentiate into all types of somatic cells, and aslo solve the ethical barriers of ESc. Induced pluripotent stem cells are not only valuable for cell replacement therapy and regenerative medicine, but also useful for disease modeling in vitro, facilitating studies of mechanisms underlying disease development, drug screening, and development of new therapeutic strategy. Although, the morbidity of neural hereditary diseases ranks first over all hereditary diseases, their pathogenesis remains unclear till now. Patient-specific iPSCs have been gradually applied as in vitro models for the study of the pathogenesis of neural hereditary diseases, drug screening as well as clinical applications. In recent years, the iPS cells models from patients with Parkinson＇s disease, Alzheimer＇s disease, amyotrophic lateral sclerosis, Huntington＇s disease and spinal muscular atrophy have been applied to explore the molecular mechanism of these diseases. Here, we review the developing course of iPSc, methods optimization of induced iPS cells to prevent virus gene interference, and the stem cells especially the iPS cell transplantation therapy of nervous system diseases such as Parkinson＇s disease, the present situation and application prospects of iPSCs.
出处
《现代生物医学进展》
CAS
2015年第12期2370-2374,共5页
Progress in Modern Biomedicine
基金
国家自然科学基金项目(81301333)
安徽省自然科学重点项目(KJ2012A199)
安徽省自然科学基金项目(10040606Q43)
国家级大学生创新创业训练计划项目(201210367021)
关键词
诱导型多能干细胞
体细胞重编程
帕金森综合症
Induced pluripotent stem cells （iPS cells）
Somatic cell reprogramming
Parkinson＇s disease
