Yates F, Daley G Q
Division of Hematology/Oncology, Children's Hospital, Boston, MA 02115, USA.
Gene Ther. 2006 Oct;13(20):1431-9. doi: 10.1038/sj.gt.3302854.
With the isolation of human embryonic stem cells (hESCs) in 1998 came the realization of a long-sought aspiration for an unlimited source of human tissue. The difficulty of differentiating ESCs to pure, clinically exploitable cell populations to treat genetic and degenerative diseases is being solved in part with the help of genetically modified cell lines. With progress in genome editing and somatic cell nuclear transfer, it is theoretically possible to obtain genetically repaired isogenic cells. Moreover, the prospect of being able to select, isolate and expand a single cell to a vast population of cells could achieve a unique level of quality control, until now unattainable in the field of gene therapy. Most of the tools necessary to develop these strategies already exist in the mouse ESC system. We review here the advances accomplished in those fields and present some possible applications to hESC research.
1998年人类胚胎干细胞（hESC）的分离实现了人们长期以来对无限人类组织来源的追求。在基因改造细胞系的帮助下，部分解决了将胚胎干细胞分化为纯净的、可用于临床治疗遗传和退行性疾病的细胞群体的难题。随着基因组编辑和体细胞核移植技术的进步，从理论上讲，有可能获得基因修复的同基因细胞。此外，能够将单个细胞选择、分离并扩增为大量细胞的前景，可以实现前所未有的质量控制水平，这在基因治疗领域是目前无法实现的。开发这些策略所需的大多数工具已存在于小鼠胚胎干细胞系统中。我们在此回顾这些领域取得的进展，并介绍一些对人类胚胎干细胞研究可能的应用。
