CRISPR GENOME SURGERY IN THE RETINA IN LIGHT OF OFF-TARGETING.
CRISPR GENOME SURGERY IN THE RETINA IN LIGHT OF OFF-TARGETING.
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DOI：
10.1097/iae.0000000000002197
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发表时间：
2018-08
期刊：
影响因子：
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通讯作者：
Mahajan VB
中科院分区：
文献类型：
--
作者：
Cho GY;Schaefer KA;Bassuk AG;Tsang SH;Mahajan VB
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. As CRISPR utilization in the laboratory setting has increased, as well as the knowledge regarding CRISPR mechanisms including its off-target effects. Although a perfect method for achieving 100% specificity has yet to be determined, the last few years has seen many developments in off-targeting detection and in increasing CRISPR tools efficacy. 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.
