Application of CRISPR-Cas9 in eye disease

被引:8
|
作者
Wu, Wenyi [1 ,2 ]
Tang, Luosheng [2 ]
D'Amore, Patricia A. [1 ,3 ]
Lei, Hetian [1 ]
机构
[1] Harvard Med Sch, Schepens Eye Res Inst, Massachusetts Eye & Ear, Dept Ophthalmol, 20 Staniford St, Boston, MA 02114 USA
[2] Cent S Univ, Xiangya Hosp 2, Dept Ophthalmol, Changsha, Hunan, Peoples R China
[3] Harvard Med Sch, Dept Pathol, Boston, MA 02114 USA
基金
美国国家卫生研究院;
关键词
STRAND BREAK REPAIR; COMPLETE GENOME SEQUENCE; PLURIPOTENT STEM-CELLS; GENE-THERAPY; HOMOLOGOUS RECOMBINATION; CONDITIONAL ALLELES; CAS SYSTEMS; RNA; CRISPR/CAS9; NUCLEASES;
D O I
10.1016/j.exer.2017.06.007
中图分类号
R77 [眼科学];
学科分类号
100212 ;
摘要
The system of clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated nuclease (Cas)9 is an effective instrument for revising the genome with great accuracy. This system has been widely employed to generate mutants in genomes from plants to human cells. Rapid improvements in Cas9 specificity in eukaryotic cells have opened great potential for the use of this technology as a therapeutic. Herein, we summarize the recent advancements of CRISPR-Cas9 use in research on human cells and animal models, and outline a basic and clinical pipeline for CRISPR-Cas9-based treatments of genetic eye diseases. (C) 2017 Elsevier Ltd. All rights reserved.
引用
收藏
页码:116 / 123
页数:8
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