Laboratory Medicine, University of California, San Francisco, California, USA.
Clin Transl Sci. 2011 Aug;4(4):298-305. doi: 10.1111/j.1752-8062.2011.00281.x.
Human embryonic stem cells (hESCs) hold great promise for ushering in an era of novel cell therapies to treat a wide range of rare and common diseases, yet they also provide an unprecedented opportunity for basic research to yield clinical benefit. HESCs can be used to better understand human development, to model human diseases, to understand the contribution of specific mutations to the pathogenesis of disease, and to develop human cell-based screening systems to identify novel therapeutic agents and evaluate potential toxicity of therapeutic agents under development. Such basic research will benefit greatly from efficient methods to perform targeted gene modification, an area of hESC investigation that is currently in its infancy. Moreover, the reality of hESC-based cellular therapies will require improved methods for generating the specific cells of interest, and reporter cell lines generated through targeted gene modifications are expected to play an important role in developing optimal cell-specific differentiation protocols. Herein, we review the current status of homologous recombination in hESCs, a gene targeting technique that is sure to continue to improve, and to play an important role in realizing the maximal human benefit from hESCs.
人胚胎干细胞（hESCs）有望开创细胞疗法的新时代，用于治疗广泛的罕见和常见疾病，但它们也为基础研究提供了一个前所未有的机会，以产生临床效益。hESCs 可用于更好地理解人类发育，模拟人类疾病，了解特定突变对疾病发病机制的贡献，以及开发基于人类细胞的筛选系统，以鉴定新的治疗剂，并评估开发中的治疗剂的潜在毒性。这种基础研究将从高效的靶向基因修饰方法中获益匪浅，而 hESC 研究领域目前仍处于起步阶段。此外，基于 hESC 的细胞疗法的现实情况需要改进生成特定目标细胞的方法，而通过靶向基因修饰产生的报告细胞系有望在开发最佳细胞特异性分化方案方面发挥重要作用。在此，我们综述 hESCs 中同源重组的现状，这是一种基因靶向技术，肯定会不断改进，并在从 hESCs 中实现最大的人类获益方面发挥重要作用。
