Progress in Targeted
Gene Integration
LI Zhu-Hong, LIU De-Pei* and LIANG Chih-Chuan
(National laboratory of Medical Molecular Biology, Institute of Basic
Medical Sc iences,
Chinese Academy of Medical Sciences & Peking Union Medical College ,
Beijing 100005, China )
Abstract The ultimate goal of gene
therapy is correction of the genetic disor ders by introducing the genetic
materials into targeted cells, and gene expressi on 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, becau se the gene
expression is controlled by the natural elements. The progress in ho mologous
recombination recently made it possible to correct the genetic disorder s by
targeted integration of the interest gene.
Key Words Gene therapy； integration； homologous
recombination
Corresponding author:
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