CRISPR基因手术用于视网膜疾病的床边治疗翻译
Translation of CRISPR Genome Surgery to the Bedside for Retinal Diseases.
作者信息
Xu Christine L, Cho Galaxy Y, Sengillo Jesse D, Park Karen S, Mahajan Vinit B, Tsang Stephen H
机构信息
Jonas Children's Vision Care, Bernard & Shirlee Brown Glaucoma Laboratory, Columbia University, New York, NY, United States.
Department of Ophthalmology, Columbia University, New York, NY, United States.
出版信息
Front Cell Dev Biol. 2018 May 23;6:46. doi: 10.3389/fcell.2018.00046. eCollection 2018.
In recent years, there has been accelerated growth of clustered regularly interspaced short palindromic repeats (CRISPR) genome surgery techniques. Genome surgery holds promise for diseases for which a cure currently does not exist. In the field of ophthalmology, CRISPR offers possibilities for treating inherited retinal dystrophies. The retina has little regenerative potential, which makes treatment particularly difficult. For such conditions, CRISPR genome surgery methods have shown great potential for therapeutic applications in animal models of retinal dystrophies. Much anticipation surrounds the potential for CRISPR as a therapeutic, as clinical trials of ophthalmic genome surgery are expected to begin as early as 2018. This mini-review summarizes preclinical CRISPR applications in the retina and current CRISPR clinical trials.
近年来，成簇规律间隔短回文重复序列（CRISPR）基因组手术技术发展迅速。基因组手术有望治愈目前尚无有效疗法的疾病。在眼科领域，CRISPR为治疗遗传性视网膜营养不良提供了可能。视网膜的再生潜力很小，这使得治疗尤为困难。对于此类病症，CRISPR基因组手术方法在视网膜营养不良动物模型的治疗应用中已显示出巨大潜力。人们对CRISPR作为一种治疗手段的潜力寄予厚望，因为眼科基因组手术临床试验预计最早将于2018年开始。本综述总结了CRISPR在视网膜的临床前应用以及当前的CRISPR临床试验。
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