Department of Pediatrics, Stanford University School of Medicine, Stanford, CA 94305, USA.
Curr Opin Immunol. 2012 Oct;24(5):609-16. doi: 10.1016/j.coi.2012.08.005. Epub 2012 Sep 13.
Genome engineering is an emerging strategy to treat monogenic diseases that relies on the use of engineered nucleases to correct mutations at the nucleotide level. Zinc finger nucleases can be designed to stimulate homologous recombination-mediated gene targeting at a variety of loci, including genes known to cause the primary immunodeficiencies (PIDs). Recently, these nucleases have been used to correct disease-causing mutations in human cells, as well as to create new animal models for human disease. Although a number of hurdles remain before they can be used clinically, engineered nucleases hold increasing promise as a therapeutic tool, particularly for the PIDs.
基因组编辑是一种新兴的治疗单基因疾病的策略，依赖于工程化核酸酶在核苷酸水平上纠正突变。锌指核酸酶可以被设计用于刺激同源重组介导的基因靶向，靶向的位点包括已知导致原发性免疫缺陷病（PID）的基因。最近，这些核酸酶已被用于纠正人类细胞中的致病突变，以及创建用于人类疾病的新型动物模型。尽管在临床应用之前仍存在许多障碍，但工程化核酸酶作为一种治疗工具具有越来越大的潜力，特别是对于 PID。
