Crook Jeremy Micah, Kobayashi Nao Rei
Stem Cell Disease Models Laboratory, Institute of Medical Biology, Singapore 138648, Singapore.
J Cell Biochem. 2008 Dec 15;105(6):1361-6. doi: 10.1002/jcb.21967.
The availability of human stem cells heralds a new era for modeling normal and pathologic tissues and developing therapeutics. For example, the in vitro recapitulation of normal and aberrant neurogenesis holds significant promise as a tool for de novo modeling of neurodevelopmental and neurodegenerative diseases. Translational applications include deciphering brain development, function, pathologies, traditional medications, and drug discovery for novel pharmacotherapeutics. For the latter, human stem cell-based assays represent a physiologically relevant and high-throughput means to assess toxicity and other undesirable effects early in the drug development pipeline, avoiding late-stage attrition whilst expediting proof-of-concept of genuine drug candidates. Here we consider the potential of human embryonic, adult, and induced pluripotent stem cells for studying neurological disorders and preclinical drug development.
人类干细胞的可得性预示着一个用于构建正常和病理组织以及开发治疗方法的新时代。例如，正常和异常神经发生的体外重现作为一种用于从头构建神经发育和神经退行性疾病模型的工具具有重大前景。转化应用包括解读大脑发育、功能、病理学、传统药物以及新型药物治疗的药物发现。对于后者，基于人类干细胞的检测代表了一种生理相关且高通量的方法，可在药物开发流程的早期评估毒性和其他不良影响，避免后期淘汰，同时加快真正候选药物的概念验证。在此，我们探讨人类胚胎干细胞、成体干细胞和诱导多能干细胞在研究神经疾病和临床前药物开发方面的潜力。
