Sangamo BioSciences Inc., Richmond, California 94804, USA.
Nat Rev Genet. 2010 Sep;11(9):636-46. doi: 10.1038/nrg2842.
Reverse genetics in model organisms such as Drosophila melanogaster, Arabidopsis thaliana, zebrafish and rats, efficient genome engineering in human embryonic stem and induced pluripotent stem cells, targeted integration in crop plants, and HIV resistance in immune cells - this broad range of outcomes has resulted from the application of the same core technology: targeted genome cleavage by engineered, sequence-specific zinc finger nucleases followed by gene modification during subsequent repair. Such 'genome editing' is now established in human cells and a number of model organisms, thus opening the door to a range of new experimental and therapeutic possibilities.
在模式生物如黑腹果蝇、拟南芥、斑马鱼和大鼠中进行反向遗传学，在人类胚胎干细胞和诱导多能干细胞中进行高效的基因组工程，在作物植物中进行靶向整合，以及在免疫细胞中实现 HIV 抗性——这些广泛的结果都源于同一核心技术的应用：通过工程化的、序列特异性的锌指核酸酶进行靶向基因组切割，然后在随后的修复过程中进行基因修饰。这种“基因组编辑”现在已经在人类细胞和一些模式生物中建立起来，从而为一系列新的实验和治疗可能性打开了大门。
