锌指核酸酶在基因组定向修饰中的应用

同源重组和逆转录病毒介导转基因法是目前基因组修饰中常用的两种主要方法.由于这些传统方法效率低，特异性差等缺点，制约了其在研究中的应用.锌指核酸酶(zinc finger nuclease，ZFN）是一种人工合成酶，含有锌指蛋白DNA结合域和非特异性核酸酶FokI结构域. ZFN在对基因组的靶向修饰时，表现出高度特异性和高效性. 最新研究结果显示，锌指核酸酶在哺乳动物细胞和斑马鱼基因组靶向敲除的效率高达20%.这一技术的出现，将给基因组靶向修饰的研究和应用领域带来革命，特别是在基因治疗人类疾病方面有巨大的潜力和广阔的前景.

Application of Zinc Finger Nuclease in Genome Targeting Modification

The homologous recombination and viral-mediated gene transfer are two commonly used methods in animal genome engineering.The extreme low efficiency and poor specificity hampered their application in generation of transgenic animals and human gene therapy.The newly developed technology,zinc finger nucleases(ZFNs) targeted genome engineering,shows promise in improving the efficiency of gene targeting by introducing DNA double-strand breaks(DSB) in target genes,which then stimulate the cell's endogenous homolo...