Therapeutic genome editing: prospects and challenges

被引：962

作者：

Cox, David Benjamin Turitz ^{[1
,2
,3
,4
]}

Platt, Randall Jeffrey ^{[1
,4
,5
,6
]}

Zhang, Feng ^{[1
,4
,5
,6
]}

机构：

[1] Broad Inst Harvard & MIT, Cambridge, MA 02142 USA

[2] MIT, Div Hlth Sci & Technol, Cambridge, MA 02139 USA

[3] MIT, Dept Biol, Cambridge, MA USA

[4] MIT, McGovern Inst Brain Res, Cambridge, MA 02139 USA

[5] MIT, Dept Brain & Cognit Sci, Cambridge, MA 02139 USA

[6] MIT, Dept Biol Engn, Cambridge, MA 02139 USA

来源：

NATURE MEDICINE | 2015年 / 21卷 / 02期

基金：

美国国家科学基金会; 美国国家卫生研究院;

关键词：

ZINC-FINGER NUCLEASES; STRAND BREAK REPAIR; SEVERE COMBINED IMMUNODEFICIENCY; DNA-BINDING SPECIFICITY; GENE-THERAPY; IN-VIVO; HOMOLOGOUS RECOMBINATION; IMMUNE-RESPONSES; T-CELLS; HOMING ENDONUCLEASES;

D O I：

10.1038/nm.3793

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：

Recent advances in the development of genome editing technologies based on programmable nucleases have substantially improved our ability to make precise changes in the genomes of eukaryotic cells. Genome editing is already broadening our ability to elucidate the contribution of genetics to disease by facilitating the creation of more accurate cellular and animal models of pathological processes. A particularly tantalizing application of programmable nucleases is the potential to directly correct genetic mutations in affected tissues and cells to treat diseases that are refractory to traditional therapies. Here we discuss current progress toward developing programmable nuclease-based therapies as well as future prospects and challenges.

引用

页码：121 / 131

页数：11

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