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CRISPR applications in ophthalmologic genome surgery

Cabral, Thiago; DiCarlo, James E.; Justus, Sally; Sengillo, Jesse D.; Xu, Yu; Tsang, Stephen H.

Current Opinion in Ophthalmology: May 2017 - Volume 28 - Issue 3 - p 252–259
doi: 10.1097/ICU.0000000000000359
TRANSLATIONAL RESEARCH: Edited by Jason Hsu and Sunir Garg
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Purpose of review The present review seeks to summarize and discuss the application of clustered regularly interspaced short palindromic repeats (CRISPR)-associated systems (Cas) for genome editing, also called genome surgery, in the field of ophthalmology.

Recent findings Precision medicine is an emerging approach for disease treatment and prevention that takes into account the variability of an individual's genetic sequence. Various groups have used CRISPR-Cas genome editing to make significant progress in mammalian preclinical models of eye disease, the basic science of eye development in zebrafish, the in vivo modification of ocular tissue, and the correction of stem cells with therapeutic applications. In addition, investigators have creatively used the targeted mutagenic potential of CRISPR-Cas systems to target pathogenic alleles in vitro.

Summary Over the past year, CRISPR-Cas genome editing has been used to correct pathogenic mutations in vivo and in transplantable stem cells. Although off-target mutagenesis remains a concern, improvement in CRISPR-Cas technology and careful screening for undesired mutations will likely lead to clinical eye therapeutics employing CRISPR-Cas systems in the near future.

aJonas Children's Vision Care, and Bernard & Shirlee Brown Glaucoma Laboratory, Department of Ophthalmology, Columbia University Medical Center

bEdward S Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY, USA

cDepartment of Ophthalmology, Federal University of Espírito Santo, Vitoria, Brazil

dDepartment of Ophthalmology, Federal University of São Paulo, São Paulo, Brazil

eState University of New York Downstate Medical Center, Brooklyn, NY, USA

fDepartment of Ophthalmology, Xin Hua Hospital Affiliated to Shanghai Jiao Tong University, Shanghai, China

gDepartment of Pathology & Cell Biology, Institute of Human Nutrition, College of Physicians and Surgeons, Columbia University, New York, NY, USA

Correspondence to Stephen H. Tsang, Jonas Children's Vision Care and Bernard & Shirlee Brown Glaucoma Laboratory, Department of Ophthalmology, Columbia University Medical Center & the Edward S Harkness Eye Institute, Columbia University Medical Center, 635 West 165th Street, 5th floor, New York, NY 10032. Tel: +1 212 342 1189; fax: +1 212 305 4987; e-mail: sht2@columbia.edu

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