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基因治疗的目的是将遗传物质导入细胞并使之得到适宜水平的表达，以纠正机体的遗传缺陷，恢复细胞的正常功能或杀死癌细胞及致病微生物。目前广泛应用的病毒及非病毒载体不能很好地满足临床要求，是基因治疗亟需解决的关键技术。同源重组介导的基因靶向性整合，是遗传性疾病基因治疗的较佳方案。近年来有关同源重组研究的进展，使得其应用于基因治疗成为可能。
The purpose of gene therapy is to introduce genetic material into cells and to obtain the appropriate level of expression to correct genetic defects in the body, to restore the normal function of cells or to kill cancer cells and pathogenic microorganisms. Currently widely used viruses and non-viral vectors can not meet the clinical requirements well and are the key technologies that need to be solved in gene therapy. Homologous recombination-mediated gene-targeted integration is a better option for gene therapy of genetic diseases. In recent years, the progress of homologous recombination research makes it possible to apply it to gene therapy.