TALEN or Cas9-Rapid,Efficient and Specific Choices for Genome Modifications  

作　　者：Chuanxian Wei Jiyong Liu Zhongsheng Yu Bo Zhang Guanjun Gao Renjie Jiao 

机构地区：[1]State Key Laboratory of Brain and Cognitive Science,Institute of Biophysics,Chinese Academy of Sciences [2]University of Chinese Academy of Sciences [3]College of Life Sciences,Peking University [4]College of Life Sciences,Tsinghua University

出　　处：《Journal of Genetics and Genomics》2013年第6期281-289,共9页遗传学报（英文版）

基　　金：supported financially by the National Basic Research Program of China(973 Program)(Nos. 2009CB918702 and 2012CB825504);the National Natural Science Foundation of China(Nos.31201007,31271573 and 31071087)

摘　　要：Precise modifications of complex genomes at the single nucleotide level have been one of the big goals for scientists working in basic and applied genetics,including biotechnology,drug development,gene therapy and synthetic biology.However,the relevant techniques for making these manipulations in model organisms and human cells have been lagging behind the rapid high throughput studies in the post-genomic era with a bottleneck of low efficiency,time consuming and laborious manipulation,and off-targeting problems.Recent discoveries of TALEs（transcription activator-like effectors） coding system and CRISPR（clusters of regularly interspaced short palindromic repeats） immune system in bacteria have enabled the development of customized TALENs（transcription activator-like effector nucleases） and CRISPR/Cas9 to rapidly edit genomic DNA in a variety of cell types,including human cells,and different model organisms at a very high efficiency and specificity.In this review,we first briefly summarize the development and applications of TALENs and CRISPR/Cas9-mediated genome editing technologies;compare the advantages and constraints of each method;particularly,discuss the expected applications of both techniques in the field of site-specific genome modification and stem cell based gene therapy;finally, propose the future directions and perspectives for readers to make the choices.Precise modifications of complex genomes at the single nucleotide level have been one of the big goals for scientists working in basic and applied genetics,including biotechnology,drug development,gene therapy and synthetic biology.However,the relevant techniques for making these manipulations in model organisms and human cells have been lagging behind the rapid high throughput studies in the post-genomic era with a bottleneck of low efficiency,time consuming and laborious manipulation,and off-targeting problems.Recent discoveries of TALEs（transcription activator-like effectors） coding system and CRISPR（clusters of regularly interspaced short palindromic repeats） immune system in bacteria have enabled the development of customized TALENs（transcription activator-like effector nucleases） and CRISPR/Cas9 to rapidly edit genomic DNA in a variety of cell types,including human cells,and different model organisms at a very high efficiency and specificity.In this review,we first briefly summarize the development and applications of TALENs and CRISPR/Cas9-mediated genome editing technologies;compare the advantages and constraints of each method;particularly,discuss the expected applications of both techniques in the field of site-specific genome modification and stem cell based gene therapy;finally, propose the future directions and perspectives for readers to make the choices.

关 键 词：Genome editing TALEN CRISPR/Cas9 Gene therapy Stem cells 

分 类 号：Q78[生物学—分子生物学]