CRISPR 基因组编辑技术在视网膜中的应用：脱靶效应的相关考量
CRISPR GENOME SURGERY IN THE RETINA IN LIGHT OF OFF-TARGETING.
机构信息
Institute of Human Nutrition, Columbia Stem Cell Initiative, College of Physicians and Surgeons, Columbia University, New York, New York.
Jonas Children's Vision Care, Bernard and Shirlee Brown Glaucoma Laboratory, New York, New York.
出版信息
Retina. 2018 Aug;38(8):1443-1455. doi: 10.1097/IAE.0000000000002197.
PURPOSE
Recent concerns regarding the clinical utilization of clustered regularly interspaced short palindromic repeats (CRISPR) involve uncertainties about the potential detrimental effects that many arise due to unintended genetic changes, as in off-target mutagenesis, during CRISPR genome surgery. This review gives an overview of off-targeting detection methods and CRISPR's place in the clinical setting, specifically in the field of ophthalmology.
RESULTS
As CRISPR utilization in the laboratory setting has increased, knowledge regarding CRISPR mechanisms including its off-target effects has also increased. Although a perfect method for achieving 100% specificity is yet to be determined, the past few years have seen many developments in off-targeting detection and in increasing efficacy of CRISPR tools.
CONCLUSION
The CRISPR system has high potential to be an invaluable therapeutic tool as it has the ability to modify and repair pathogenic retinal lesions. Although it is not yet a perfect system, with further efforts to improve its specificity and efficacy along with careful screening of off-target mutations, CRISPR-mediated genome surgery potential can become maximized and applied to patients.
目的
最近人们对簇状规律间隔短回文重复序列（CRISPR）的临床应用产生了一些担忧，主要是因为在 CRISPR 基因组手术过程中，许多非预期的基因变化（如脱靶诱变）可能会产生潜在的有害影响。本综述概述了脱靶检测方法以及 CRISPR 在临床环境中的应用，特别是在眼科领域的应用。
结果
随着 CRISPR 在实验室中的应用不断增加，人们对 CRISPR 机制（包括其脱靶效应）的了解也不断增加。尽管尚未确定实现 100%特异性的完美方法，但在过去几年中，脱靶检测和提高 CRISPR 工具效率方面取得了许多进展。
结论
CRISPR 系统具有成为一种非常有价值的治疗工具的巨大潜力，因为它能够修饰和修复致病性视网膜病变。尽管它还不是一个完美的系统，但通过进一步努力提高其特异性和效率，并仔细筛选脱靶突变，CRISPR 介导的基因组手术潜力可以得到最大化，并应用于患者。
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