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Genome engineering of human pluripotent stem cells and mice using site-specific nucleases

日期: 2013-05-09
学术报告
题目:Genome engineering of human pluripotent stem cells and mice using site-specific nucleases
报告人:Haoyi Wang
Postdoctoral Associate, Laboratory of Dr. Rudolf Jaenisch,
Whitehead Institute for Biomedical Research,
Massachusetts Institute of Technology
时间:2013年5月13日(周一),9:30-10:30 AM
地点:生命科学学院345室
Targeted genetic engineering of mice and human pluripotent stem cells is essential for understanding gene function in development and disease. Gene targeting of human pluripotent stem cells by homologous recombination is inefficient which has impeded the use of human ES and iPS cells in disease models.
Using Transcription activator-like effector nucleases (TALENs), we developed strategies to efficiently modify genome of human pluripotent cells, which allows for establishment of novel disease models and screen platforms. In addition, we produced the world first mice harboring targeted mutations and insertions in Y-linked genes, providing a novel and general approach for genetic manipulation of the Y chromosome, which has not been possible with conventional gene targeting approaches.
Furthermore, using CRISPR/Cas system we achieved multiplexed genome editing in mES cells and mouse with high efficiency. This method allows the one step generation of animals carrying mutations in multiple genes, an approach that will greatly accelerate the in vivo study of functionally redundant genes and of epistatic gene interactions.
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