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对CRISPR-Cas系统的天然调控机制及临床应用的洞察。

Insight into the natural regulatory mechanisms and clinical applications of the CRISPR-Cas system.

作者信息

Cheng Hui, Deng Haoyue, Ma Dongdao, Gao Mengyuan, Zhou Zhihan, Li Heng, Liu Shejuan, Teng Tieshan

机构信息

School of Basic Medical Sciences, Henan University, Kaifeng, 475004, China.

School of Medical Laboratory, Weifang Medical University, Weifang, 261053, Shandong, China.

出版信息

Heliyon. 2024 Oct 18;10(20):e39538. doi: 10.1016/j.heliyon.2024.e39538. eCollection 2024 Oct 30.

Abstract

CRISPR-Cas, the adaptive immune system exclusive to prokaryotes, confers resistance against foreign mobile genetic elements. The CRISPR-Cas system is now being exploited by scientists in a diverse range of genome editing applications. CRISPR-Cas systems can be categorized into six different types based on their composition and mechanism, and there are also natural regulatory biomolecules in bacteria and bacteriophages that can either enhance or inhibit the immune function of CRISPR-Cas. The CRISPR-Cas systems are currently being trialed as a new tool for gene therapy to treat various human diseases, including cancers and genetic diseases, offering significant therapeutic potential. This paper comprehensively summarizes various aspects of the CRISPR-Cas system, encompassing its diversity, regulatory mechanisms, its clinical applications and the obstacles encountered.

摘要

CRISPR-Cas是原核生物特有的适应性免疫系统,可抵御外来可移动遗传元件。目前,科学家们正在将CRISPR-Cas系统应用于各种基因组编辑领域。根据其组成和作用机制,CRISPR-Cas系统可分为六种不同类型,细菌和噬菌体中也存在天然调节生物分子,它们可以增强或抑制CRISPR-Cas的免疫功能。目前,CRISPR-Cas系统正作为一种新的基因治疗工具进行试验,用于治疗包括癌症和遗传疾病在内的各种人类疾病,具有巨大的治疗潜力。本文全面总结了CRISPR-Cas系统的各个方面,包括其多样性、调节机制、临床应用及遇到的障碍。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/386e/11535992/728c4b516155/gr1.jpg

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