Advances in targeted genome editing

被引:107
作者
Perez-Pinera, Pablo [1 ]
Ousterout, David G. [1 ]
Gersbach, Charles A. [1 ]
机构
[1] Duke Univ, Dept Biomed Engn, Durham, NC 27708 USA
基金
美国国家科学基金会;
关键词
ZINC-FINGER NUCLEASES; PLURIPOTENT STEM-CELLS; DOUBLE-STRAND BREAKS; GENE DISRUPTION; IN-VIVO; HOMOLOGOUS RECOMBINATION; DIRECTED EVOLUTION; CHIMERIC NUCLEASES; NICKING ENZYME; VIRAL VECTORS;
D O I
10.1016/j.cbpa.2012.06.007
中图分类号
Q5 [生物化学]; Q7 [分子生物学];
学科分类号
071010 ; 081704 ;
摘要
New technologies have recently emerged that enable targeted editing of genomes in diverse systems. This includes precise manipulation of gene sequences in their natural chromosomal context and addition of transgenes to specific genomic loci. This progress has been facilitated by advances in engineering targeted nucleases with programmable, site-specific DNA-binding domains, including zinc finger proteins and transcription activator-like effectors (TALEs). Recent improvements have enhanced nuclease performance, accelerated nuclease assembly, and lowered the cost of genome editing. These advances are driving new approaches to many areas of biotechnology, including biopharmaceutical production, agriculture, creation of transgenic organisms and cell lines, and studies of genome structure, regulation, and function. Genome editing is also being investigated in preclinical and clinical gene therapies for many diseases.
引用
收藏
页码:268 / 277
页数:10
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