CRISPR-Cas Genome Surgery in Ophthalmology

被引：12

作者：

DiCarlo, James E. ^{[1
,2
,3
]}

Sengillo, Jesse D. ^{[1
,2
,3
,4
]}

Justus, Sally ^{[1
,2
,3
]}

Cabral, Thiago ^{[1
,2
,3
,5
,6
]}

Tsang, Stephen H. ^{[1
,2
,3
,7
]}

Mahajan, Vinit B. ^{[8
,9
]}

机构：

[1] Columbia Univ, Dept Ophthalmol, Jonas Childrens Vis Care, New York, NY 10027 USA

[2] Columbia Univ, Dept Ophthalmol, Bernard & Shirlee Brown Glaucoma Lab, New York, NY 10027 USA

[3] New York Presbyterian Hosp, Edward S Harkness Eye Inst, New York, NY 10032 USA

[4] Suny Downstate Med Ctr, Brooklyn, NY 11203 USA

[5] Univ Fed Espirito Santo, Dept Ophthalmol, Vitoria, Brazil

[6] Univ Fed Sao Paulo, Dept Ophthalmol, Sao Paulo, Brazil

[7] Columbia Univ, Coll Phys & Surg, Inst Human Nutr, Dept Pathol & Cell Biol, New York, NY USA

[8] Stanford Univ, Dept Ophthalmol, Byers Eye Inst, Omics Lab, Palo Alto, CA 94304 USA

[9] Stanford Univ, Byers Eye Inst, Dept Ophthalmol, Palo Alto, CA 94304 USA

来源：

TRANSLATIONAL VISION SCIENCE & TECHNOLOGY | 2017年 / 6卷 / 03期

基金：

美国国家卫生研究院;

关键词：

CRISPR-Cas; genome engineering; gene therapy; synthetic biology; precision medicine; PLURIPOTENT STEM-CELLS; RETINAL GANGLION-CELLS; DOUBLE-STRAND BREAKS; HUMAN IPS CELLS; GENE-THERAPY; HOMOLOGOUS RECOMBINATION; CONGENITAL AMAUROSIS; SOMATIC MOSAICISM; MAMMALIAN-CELLS; DNA;

D O I：

10.1167/tvst.6.3.13

中图分类号：

R77 [眼科学];

学科分类号：

100212 ;

摘要：

Genetic disease affecting vision can significantly impact patient quality of life. Gene therapy seeks to slow the progression of these diseases by treating the underlying etiology at the level of the genome. Clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated systems (Cas) represent powerful tools for studying diseases through the creation of model organisms generated by targeted modification and by the correction of disease mutations for therapeutic purposes. CRISPR-Cas systems have been applied successfully to the visual sciences and study of ophthalmic disease -from the modification of zebrafish and mammalian models of eye development and disease, to the correction of pathogenic mutations in patientderived stem cells. Recent advances in CRISPR-Cas delivery and optimization boast improved functionality that continues to enhance genome-engineering applications in the eye. This review provides a synopsis of the recent implementations of CRISPRCas tools in the field of ophthalmology.

引用

页数：13

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