Szebényi Kornélia, Erdei Zsuzsa, Péntek Adrienn, Sebe Attila, Orbán Tamás I, Sarkadi Balázs, Apáti Ágota
Membrane Research Group, Hungarian Academy of Sciences & National Blood Center, 1113 Budapest, Diószegi u. 64, Hungary.
Department of Biochemistry & Molecular Biology, Medical & Health Science Center, University of Debrecen, Debrecen, Hungary.
Per Med. 2011 May;8(3):347-364. doi: 10.2217/pme.11.19.
Human stem cells provide an important novel tool for generating in vitro pharmacological and toxicological test systems. In the development of new targeted therapies, as well as in critical safety issues, including hepato-, neuro- and cardio-toxicity, animal-based tests are mostly unsatisfactory, whereas the use of in vitro model systems is limited by the unavailability of relevant human tissues. Human embryonic stem cell lines may fill this gap and offer an advantage over primary cultures as well as tissue-derived (adult) stem cells. Human embryonic stem cells represent an unlimited source for the production of differentiated somatic progenies and allow various stable genetic manipulations. As a new opening in personalized medicine test systems, the generation of induced pluripotent stem cell lines and their derivatives can provide patient- and disease-specific cellular assays for drug development and safety assessments. This article reviews promising human stem cell applications in pharmacological and toxicological screenings, focusing on the implications for personalized medicine.
人类干细胞为生成体外药理学和毒理学测试系统提供了一种重要的新型工具。在新的靶向治疗药物的研发过程中，以及在包括肝毒性、神经毒性和心脏毒性等关键安全性问题方面，基于动物的测试大多不尽人意，而体外模型系统的应用则因缺乏相关人类组织而受到限制。人类胚胎干细胞系可能填补这一空白，并且相较于原代培养物以及组织来源的（成体）干细胞具有优势。人类胚胎干细胞是分化体细胞后代产生的无限来源，并允许进行各种稳定的基因操作。作为个性化医学测试系统的一个新开端，诱导多能干细胞系及其衍生物的产生可为药物开发和安全性评估提供针对患者和疾病的细胞检测方法。本文综述了人类干细胞在药理学和毒理学筛选中的应用前景，重点关注其对个性化医学的意义。
