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CRISPR-Cas技术进展及其应用:革新精准医学

Advances in CRISPR-Cas technology and its applications: revolutionising precision medicine.

作者信息

Azeez Sarkar Sardar, Hamad Rahin Shareef, Hamad Bahra Kakamin, Shekha Mudhir Sabir, Bergsten Peter

机构信息

Department of Medical Laboratory Technology, Soran Technical College, Erbil Polytechnic University, Erbil, Kurdistan Region, Iraq.

Nursing Department, Soran Technical College, Erbil Polytechnic University, Erbil, Kurdistan Region, Iraq.

出版信息

Front Genome Ed. 2024 Dec 12;6:1509924. doi: 10.3389/fgeed.2024.1509924. eCollection 2024.

Abstract

CRISPR-Cas (Clustered Regularly Interspaced Short Palindromic Repeats-CRISPR-associated proteins) has undergone marked advancements since its discovery as an adaptive immune system in bacteria and archaea, emerged as a potent gene-editing tool after the successful engineering of its synthetic guide RNA (sgRNA) toward the targeting of specific DNA sequences with high accuracy. Besides its DNA editing ability, further-developed Cas variants can also edit the epigenome, rendering the CRISPR-Cas system a versatile tool for genome and epigenome manipulation and a pioneering force in precision medicine. This review explores the latest advancements in CRISPR-Cas technology and its therapeutic and biomedical applications, highlighting its transformative impact on precision medicine. Moreover, the current status of CRISPR therapeutics in clinical trials is discussed. Finally, we address the persisting challenges and prospects of CRISPR-Cas technology.

摘要

CRISPR-Cas(成簇规律间隔短回文重复序列及其相关蛋白)自作为细菌和古生菌的一种适应性免疫系统被发现以来,取得了显著进展。在成功设计出合成引导RNA(sgRNA)以高精度靶向特定DNA序列后,它成为了一种强大的基因编辑工具。除了其DNA编辑能力外,进一步开发的Cas变体还可以编辑表观基因组,使CRISPR-Cas系统成为用于基因组和表观基因组操作的通用工具,以及精准医学中的先驱力量。本文综述探讨了CRISPR-Cas技术的最新进展及其在治疗和生物医学领域的应用,强调了其对精准医学的变革性影响。此外,还讨论了CRISPR疗法在临床试验中的现状。最后,我们阐述了CRISPR-Cas技术持续存在的挑战和前景。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7961/11669675/64e8764e7446/FGEED_fgeed-2024-1509924_wc_abs.jpg

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