靶向整合研究进展

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。

Progress in Targeted Gene Integration

The ultimate goal of gene therapy is correction of the genetic disorders by introducing the genetic materials into targeted cells, and gene expression should be permanent and at an appropriate level. The widely used gene therapy vectors nowadays can not meet the demands. The targeted integration mediated by the homologous recombination is the best strategy for human gene therapy, because the gene expression is controlled by the natural elements. The progress in homologous recombination recently made it possible to correct the genetic disorders by targeted integration of the interest gene.