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用于视网膜疾病的CRISPR/Cas9基因组手术。

CRISPR/Cas9 genome surgery for retinal diseases.

作者信息

Xu Christine L, Park Karen Sophia, Tsang Stephen H

机构信息

Edward S Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY, USA; Jonas Children's Vision Care and the Bernard & Shirlee Brown Glaucoma Laboratory, Department of Ophthalmology, Columbia University, New York, NY, USA.

Edward S Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY, USA; Jonas Children's Vision Care and the Bernard & Shirlee Brown Glaucoma Laboratory, Department of Ophthalmology, Columbia University, New York, NY, USA; Department of Pathology & Cell Biology, Institute of Human Nutrition, Columbia Stem Cell Initiative, College of Physicians and Surgeons, Columbia University, New York, NY, USA.

出版信息

Drug Discov Today Technol. 2018 Aug;28:23-32. doi: 10.1016/j.ddtec.2018.05.001. Epub 2018 Jun 18.

Abstract

Retinal diseases that impair vision can impose heavy physical and emotional burdens on patients' lives. Currently, clustered regularly interspaced short palindromic repeats (CRISPR) is a prevalent gene-editing tool that can be harnessed to generate disease model organisms for specific retinal diseases, which are useful for elucidating pathophysiology and revealing important links between genetic mutations and phenotypic defects. These retinal disease models are fundamental for testing various therapies and are indispensible for potential future clinical trials. CRISPR-mediated procedures involving CRISPR-associated protein 9 (Cas9) may also be used to edit genome sequences and correct mutations. Thus, if used for future therapies, CRISPR/Cas9 genome surgery could eliminate the need for patients with retinal diseases to undergo repetitive procedures such as drug injections. In this review, we will provide an overview of CRISPR/Cas9, discuss the different types of Cas9, and compare Cas9 to other endonucleases. Furthermore, we will explore the many ways in which researchers are currently utilizing this versatile tool, as CRISPR/Cas9 may have far-reaching effects in the treatment of retinal diseases.

摘要

损害视力的视网膜疾病会给患者的生活带来沉重的身体和情感负担。目前,成簇规律间隔短回文重复序列(CRISPR)是一种普遍使用的基因编辑工具,可用于为特定视网膜疾病构建疾病模型生物,这有助于阐明病理生理学,并揭示基因突变与表型缺陷之间的重要联系。这些视网膜疾病模型是测试各种疗法的基础,对于未来潜在的临床试验不可或缺。涉及CRISPR相关蛋白9(Cas9)的CRISPR介导程序也可用于编辑基因组序列和纠正突变。因此,如果用于未来的治疗,CRISPR/Cas9基因组手术可能使视网膜疾病患者无需进行诸如药物注射等重复性程序。在这篇综述中,我们将概述CRISPR/Cas9,讨论不同类型的Cas9,并将Cas9与其他核酸内切酶进行比较。此外,我们将探讨研究人员目前利用这种多功能工具的多种方式,因为CRISPR/Cas9在视网膜疾病治疗中可能具有深远影响。

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