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CRISPR 技术在临床实践中的应用进展更新。

An Update on the Application of CRISPR Technology in Clinical Practice.

机构信息

Department of Genetics, Faculty of Basic Sciences, Shahrekord Branch, Islamic Azad University, Shahrekord, Iran.

Department of Medical Genetics and Molecular Medicine, Faculty of Medicine, Mashhad University of Medical Sciences, Mashhad, Iran.

出版信息

Mol Biotechnol. 2024 Feb;66(2):179-197. doi: 10.1007/s12033-023-00724-z. Epub 2023 Jun 3.

Abstract

The CRISPR/Cas system, an innovative gene-editing tool, is emerging as a promising technique for genome modifications. This straightforward technique was created based on the prokaryotic adaptive immune defense mechanism and employed in the studies on human diseases that proved enormous therapeutic potential. A genetically unique patient mutation in the process of gene therapy can be corrected by the CRISPR method to treat diseases that traditional methods were unable to cure. However, introduction of CRISPR/Cas9 into the clinic will be challenging because we still need to improve the technology's effectiveness, precision, and applications. In this review, we first describe the function and applications of the CRISPR-Cas9 system. We next delineate how this technology could be utilized for gene therapy of various human disorders, including cancer and infectious diseases and highlight the promising examples in the field. Finally, we document current challenges and the potential solutions to overcome these obstacles for the effective use of CRISPR-Cas9 in clinical practice.

摘要

CRISPR/Cas 系统是一种创新的基因编辑工具,正在成为一种有前途的基因组修饰技术。这种简单直接的技术是基于原核生物的适应性免疫防御机制创建的,并应用于人类疾病的研究中,证明了其巨大的治疗潜力。在基因治疗过程中,CRISPR 方法可以纠正患者基因突变,从而治疗传统方法无法治愈的疾病。然而,将 CRISPR/Cas9 引入临床将具有挑战性,因为我们仍需要提高该技术的有效性、精度和应用。在这篇综述中,我们首先描述了 CRISPR-Cas9 系统的功能和应用。接下来,我们阐述了如何将这项技术用于治疗各种人类疾病,包括癌症和传染病,并强调了该领域有前途的实例。最后,我们记录了当前的挑战和潜在的解决方案,以克服这些障碍,从而在临床实践中有效使用 CRISPR-Cas9。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3774/10239226/b334707a3c50/12033_2023_724_Fig1_HTML.jpg

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