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关于利用CRISPR/Cas9基因组编辑技术进行分子剪接的综述。

A review on molecular scissoring with CRISPR/Cas9 genome editing technology.

作者信息

Irfan Muskan, Majeed Hammad, Iftikhar Tehreema, Ravi Pritam Kumar

机构信息

Department of Biotechnology, University of Management and Technology (UMT), Lahore, Sialkot Campus, Sialkot 51310, Pakistan.

Department of Chemistry, University of Management and Technology (UMT), Lahore, Sialkot Campus, Sialkot 51310, Pakistan.

出版信息

Toxicol Res (Camb). 2024 Jul 12;13(4):tfae105. doi: 10.1093/toxres/tfae105. eCollection 2024 Aug.

Abstract

Genome editing is a technology to make specific changes in the DNA of a cell or an organism. It has significantly altered the landscape of life sciences, facilitating the establishment of exceedingly customized genetic modifications. Among various genome editing technologies, the CRISPR/Cas9 system, a specific endonuclease induces a double stranded DNA break and enabling modifications to the genome, has surfaced as a formidable and adaptable instrument. Its significance cannot be overstated, as it not only allows for the manipulation of genomes in model organisms but also holds great potential for revolutionary advances in medicine, particularly in treating genetic diseases. This review paper explores the remarkable journey of CRISPR/Cas9, its natural function, mechanisms, and transformative impact on genome editing and finally the use of artificial intelligence and other intelligent manufacturing tools used. The introduction provides the background on genome editing, emphasizing the emergence and significance of CRISPR/Cas9. Subsequent sections comprehensively elucidate its natural function, disease modeling, agriculture, and biotechnology, address therapeutic applications, and ongoing clinical trials while also discussing prospects and ethical implications. We summarized the key findings, indicating that CRISPR/Cas9 has empowered the creation of disease-specific animal models. This provides invaluable insights into pathogenic mechanisms and opens new avenues for drug discovery, reaffirming the transformative impact of CRISPR/Cas9 on genome editing. Finally we discussed the importance of continued research and collaboration for comprehensive utilization of the inherent capabilities of this molecular precision tool in shaping forthcoming advancements.

摘要

基因组编辑是一种对细胞或生物体的DNA进行特定改变的技术。它极大地改变了生命科学的格局,促进了极其定制化的基因修饰的建立。在各种基因组编辑技术中,CRISPR/Cas9系统,一种能诱导双链DNA断裂并实现基因组修饰的特定核酸内切酶,已成为一种强大且适应性强的工具。其重要性无论如何强调都不为过,因为它不仅允许对模式生物的基因组进行操作,而且在医学,特别是在治疗遗传疾病方面具有实现革命性进展的巨大潜力。这篇综述文章探讨了CRISPR/Cas9的非凡历程、其天然功能、作用机制以及对基因组编辑的变革性影响,最后还探讨了所使用的人工智能和其他智能制造工具。引言部分提供了基因组编辑的背景,强调了CRISPR/Cas9的出现及其重要性。随后的章节全面阐述了其天然功能、疾病建模、农业和生物技术、治疗应用以及正在进行的临床试验,同时也讨论了前景和伦理影响。我们总结了关键发现,表明CRISPR/Cas9已助力创建疾病特异性动物模型。这为致病机制提供了宝贵的见解,并为药物发现开辟了新途径,再次肯定了CRISPR/Cas9对基因组编辑的变革性影响。最后,我们讨论了持续研究与合作对于全面利用这一分子精准工具的内在能力以塑造未来进展的重要性。

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