Advances in the Development of Gene-Targeting Vectors to Increase the Efficiency of Genetic Modification
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Gene targeting <i>via</i> homologous recombination, albeit highly inefficient in human cells, is considered a powerful tool for analyzing gene functions. Despite recent progress in the application of artificial nucleases for genome editing, safety issues remain a concern, particularly when genetic modification is used for therapeutic purposes. Therefore, the development of gene-targeting vectors is necessary for safe and sophisticated genetic modification. In this paper, we describe the effect of vector structure on random integration, which is a major obstacle in efficient gene targeting. In addition, we focus on the features of exon-trapping-type gene-targeting vectors, and discuss a novel strategy for negative selection to enhance gene targeting in human cells.
- Biological and Pharmaceutical Bulletin
Biological and Pharmaceutical Bulletin 39(1), 25-32, 2016
The Pharmaceutical Society of Japan