Target Discovery for Precision Medicine Using High-Throughput Genome Engineering

被引：7

作者：

Guo, Xinyi ^{[1
,2
]}

Chitale, Poonam ^{[1
,2
]}

Sanjana, Neville E. ^{[1
,2
]}

机构：

[1] New York Genome Ctr, 101 Ave Amer, New York, NY 10013 USA

[2] NYU, Dept Biol, New York, NY 10003 USA

来源：

PRECISION MEDICINE, CRISPR, AND GENOME ENGINEERING: MOVING FROM ASSOCIATION TO BIOLOGY AND THERAPEUTICS | 2017年 / 1016卷

关键词：

Genome engineering; Pooled CRISPR screens; Functional genomics; Cancer; Drug resistance; Infectious disease; Metabolism; Target identification; WIDE CRISPR SCREEN; TRANSCRIPTIONAL ACTIVATION; REGULATORY ELEMENTS; DNA METHYLATION; GENETIC SCREENS; HOST FACTORS; HUMAN-CELLS; WEST-NILE; SYSTEM; CAS9;

D O I：

10.1007/978-3-319-63904-8_7

中图分类号：

Q [生物科学];

学科分类号：

07 ; 0710 ; 09 ;

摘要：

Over the past few years, programmable RNA-guided nucleases such as the CRISPR/Cas9 system have ushered in a new era of precision genome editing in diverse model systems and in human cells. Functional screens using large libraries of RNA guides can interrogate a large hypothesis space to pinpoint particular genes and genetic elements involved in fundamental biological processes and diseaserelevant phenotypes. Here, we review recent high-throughput CRISPR screens (e.g. loss-of--function, gain-of-function, and targeting noncoding elements) and highlight their potential for uncovering novel therapeutic targets, such as those involved in cancer resistance to small molecular drugs and immunotherapies, tumor evolution, infectious disease, inborn genetic disorders, and other therapeutic challenges.

引用

页码：123 / 145

页数：23

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