Gupta Rajat M, Musunuru Kiran
J Clin Invest. 2014 Oct;124(10):4154-61. doi: 10.1172/JCI72992. Epub 2014 Oct 1.
The past decade has been one of rapid innovation in genome-editing technology. The opportunity now exists for investigators to manipulate virtually any gene in a diverse range of cell types and organisms with targeted nucleases designed with sequence-specific DNA-binding domains. The rapid development of the field has allowed for highly efficient, precise, and now cost-effective means by which to generate human and animal models of disease using these technologies. This review will outline the recent development of genome-editing technology, culminating with the use of CRISPR-Cas9 to generate novel mammalian models of disease. While the road to using this same technology for treatment of human disease is long, the pace of innovation over the past five years and early successes in model systems build anticipation for this prospect.
过去十年是基因组编辑技术快速创新的十年。现在研究人员有机会利用设计有序列特异性DNA结合结构域的靶向核酸酶,在多种细胞类型和生物体中操纵几乎任何基因。该领域的快速发展使得利用这些技术生成人类和动物疾病模型有了高效、精确且如今具有成本效益的方法。本综述将概述基因组编辑技术的最新进展,最终聚焦于使用CRISPR-Cas9生成新型哺乳动物疾病模型。虽然将这项技术用于治疗人类疾病的道路漫长,但过去五年的创新步伐以及模型系统的早期成功让人对这一前景充满期待。