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Nucleic Acids Res. 2011 Aug;39(14):6124-36. doi: 10.1093/nar/gkr186. Epub 2011 Apr 10.
Homing endonucleases (HE) have emerged as precise tools for achieving gene targeting events. Redesigned HEs with tailored specificities can be used to cleave new sequences, thereby considerably expanding the number of targetable genes and loci. With HEs, as well as with other protein scaffolds, context dependence of DNA/protein interaction patterns remains one of the major limitations for rational engineering of new DNA binders. Previous studies have shown strong crosstalk between different residues and regions of the DNA binding interface. To investigate this phenomenon, we systematically combined mutations from three groups of amino acids in the DNA binding regions of the I-CreI HE. Our results confirm that important crosstalk occurs throughout this interface in I-CreI. Detailed analysis of success rates identified a nearest-neighbour effect, with a more pronounced level of dependence between adjacent regions. Taken together, these data suggest that combinatorial engineering does not necessarily require the identification of separable functional or structural regions, and that groups of amino acids provide acceptable building blocks that can be assembled, overcoming the context dependency of the DNA binding interface. Furthermore, the present work describes a sequential method to engineer tailored HEs, wherein three contiguous regions are individually mutated and assembled to create HEs with engineered specificity.
归巢内切核酸酶 (HE) 已成为实现基因靶向事件的精确工具。经过重新设计的具有定制特异性的 HE 可用于切割新序列,从而大大增加了可靶向基因和基因座的数量。对于 HE 以及其他蛋白质支架,DNA/蛋白质相互作用模式的上下文依赖性仍然是理性设计新 DNA 结合物的主要限制因素之一。先前的研究表明,不同残基和 DNA 结合界面区域之间存在强烈的串扰。为了研究这种现象,我们系统地组合了 I-CreI HE 的 DNA 结合区域中三组氨基酸的突变。我们的结果证实,I-CreI 中整个界面都存在重要的串扰。对成功率的详细分析确定了最近邻效应,相邻区域之间的依赖性更为明显。总之,这些数据表明,组合工程不一定需要识别可分离的功能或结构区域,并且氨基酸组提供了可接受的构建块,可以进行组装,克服 DNA 结合界面的上下文依赖性。此外,本工作描述了一种工程定制 HE 的顺序方法,其中三个连续区域分别突变并组装以创建具有工程特异性的 HE。