NOVEL HUMAN GENE SPECIFIC REAGENTS FROM HOMING ENDOS

这项研究的目标是建立一个家庭， 具有修饰能力的新型、高度序列特异性的DNA结合蛋白， 人类细胞中特定基因的结构或表达。起始 基因特异性试剂（GSR）的生成点已经存在 蛋白质称为归巢核酸内切酶，可以结合和切割独特的，长 （15-40 bp）DNA靶或归巢位点，在体内具有高特异性。的 这些归巢核酸内切酶与其特异性靶标结合的机制 这表明有可能产生可以与任何靶结合GSR 具有相对高特异性的人细胞DNA序列。归巢 核酸内切酶变体，其具有修饰结构或功能的能力， 特异性人类基因将为研究人类基因提供新的机会 结构和功能。人类枪击残留物的存在也可能 对导致特定疾病或基因的人类基因的修饰 病原微生物，包括细菌和病毒、寄生虫等。

The goal of the proposed research is to generate a family of novel, highly sequence-specific DNA binding proteins with the ability to modify the structure or expression of specific genes in human cells. The starting point for the generation of the Gene-Specific-Reagents (GSR's) are existing proteins known as homing endonucleases, that can bind and cleave unique, long (15-40 bp) DNA targets or homing sites with high specificity in vivo. The mechanism by which these homing endonucleases bind to their specific targets suggest that it may be possible to generate GSR's that can bind to any target sequence of human cell DNA with relatively high specificity. Homing endonuclease variants with the ability to modify the structure or functions of specific human genes would provide new opportunities to investigate human gene structure and function. The availability of human GSR's may also allow modification of altered human genes responsible for specific diseases or genes of pathogenic microorganisms including bacteria and viruses, parasites, etc.