From Origin to the Present: Establishment, Mechanism, Evolutions and Biomedical Applications of the CRISPR/Cas-Based Macromolecular System in Brief.

IF 4.6 2区 化学 Q2 BIOCHEMISTRY & MOLECULAR BIOLOGY Molecules Pub Date : 2025-02-18 DOI:10.3390/molecules30040947
Zheng Yuan
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Abstract

Advancements in biological and medical science are intricately linked to the biological central dogma. In recent years, gene editing techniques, especially CRISPR/Cas systems, have emerged as powerful tools for modifying genetic information, supplementing the central dogma and holding significant promise for disease diagnosis and treatment. Extensive research has been conducted on the continuously evolving CRISPR/Cas systems, particularly in relation to challenging diseases, such as cancer and HIV infection. Consequently, the integration of CRISPR/Cas-based techniques with contemporary medical approaches and therapies is anticipated to greatly enhance healthcare outcomes for humans. This review begins with a brief overview of the discovery of the CRISPR/Cas system. Subsequently, using CRISPR/Cas9 as an example, a clear description of the classical molecular mechanism underlying the CRISPR/Cas system was given. Additionally, the development of the CRISPR/Cas system and its applications in gene therapy and high-sensitivity disease diagnosis were discussed. Furthermore, we address the prospects for clinical applications of CRISPR/Cas-based gene therapy, highlighting the ethical considerations associated with altering genetic information. This brief review aims to enhance understanding of the CRISPR/Cas macromolecular system and provide insight into the potential of genetic macromolecular drugs for therapeutic purposes.

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从起源到现在:基于CRISPR/ cas的大分子系统的建立、机制、演变及生物医学应用综述
生物学和医学的进步与生物学中心教条有着错综复杂的联系。近年来,基因编辑技术,特别是CRISPR/Cas系统,已经成为修改遗传信息的强大工具,补充了中心教条,并在疾病诊断和治疗方面具有重大前景。对不断发展的CRISPR/Cas系统进行了广泛的研究,特别是与癌症和艾滋病毒感染等具有挑战性的疾病有关。因此,基于CRISPR/ cas的技术与现代医学方法和疗法的整合有望大大提高人类的医疗保健结果。本综述首先简要概述了CRISPR/Cas系统的发现。随后,以CRISPR/Cas9为例,对CRISPR/Cas系统的经典分子机制进行了清晰的描述。此外,还讨论了CRISPR/Cas系统的发展及其在基因治疗和高灵敏度疾病诊断中的应用。此外,我们讨论了基于CRISPR/ cas的基因治疗的临床应用前景,强调了与改变遗传信息相关的伦理考虑。本文旨在加深对CRISPR/Cas大分子系统的认识,并为开发用于治疗目的的遗传大分子药物提供新的思路。
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来源期刊
Molecules
Molecules 化学-有机化学
CiteScore
7.40
自引率
8.70%
发文量
7524
审稿时长
1.4 months
期刊介绍: Molecules (ISSN 1420-3049, CODEN: MOLEFW) is an open access journal of synthetic organic chemistry and natural product chemistry. All articles are peer-reviewed and published continously upon acceptance. Molecules is published by MDPI, Basel, Switzerland. Our aim is to encourage chemists to publish as much as possible their experimental detail, particularly synthetic procedures and characterization information. There is no restriction on the length of the experimental section. In addition, availability of compound samples is published and considered as important information. Authors are encouraged to register or deposit their chemical samples through the non-profit international organization Molecular Diversity Preservation International (MDPI). Molecules has been launched in 1996 to preserve and exploit molecular diversity of both, chemical information and chemical substances.
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