CRISPR-Cas9技术在中药现代化中的探索
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江西中医药大学中医学院

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国家自然科学基金项目(81960808);江西省自然科学基金项目(20224BAB206029);江西省自然科学基金青年项目(20224BAB216001);江西中医药大学博士研究启动基金(020BSZR012);江西中医药大学校级科技创新团队发展计划(CXTD22013)、江西中医药大学中西医结合一级学科(江西省双一流学科)(zxyylxk20220103)资助项目。


Exploring CRISPR-Cas9 Technology in the Modernization of Traditional Chinese Medicine
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School of Traditional Chinese Medicine,Jiangxi University of Chinese Medicine

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    摘要:

    CRISPR-Cas免疫系统是一种广泛分布于细菌和古细菌,基于序列特异性的内源性免疫应答,主要用于抵御外源的噬菌体、病毒或质粒。CRISPR-Cas9系统因其卓越的修饰能力,已发展为目前基因组工程系统中应用研究最灵活高效的技术。在CRISPR-Cas9系统中,Cas9蛋白在gRNA的引导下,能够特异性识别目标DNA片段并诱导双链断裂。基于在特异性、灵活性及可操作性方面展现出的显著优势,CRISPR-Cas9技术表现出在医药领域巨大的发展前景,成为构建精确疾病模型以阐明中药复杂作用机制、促进中药改良、鉴定识别中药种质、把控中药质量和创新生产的强大工具。大量CRISPR数据同时也驱动了人工智能构建虚拟疾病模型进行药物筛选、赋能中药有效成分的发现与减毒增效,这正好满足了精准医疗的迫切需求,促进中药现代化转化进程。本文首先对CRISPR-Cas9系统的发展脉络、结构基础和运作方式进行回溯与阐释、继而系统概述精准医学与中药发展相关研究现状。在精准医疗快速发展背景下,本文重点论述 CRISPR-Cas9 基因编辑技术如何赋能中药作用机理的深入解析,并进一步阐述其在推动中药现代化进程中的具体应用。最后本文展望了CRISPR技术与人工智能相融合的发展图景:从驱动药物筛选、赋能中药有效成分发现再到中药减毒增效模型的构建,多维合力为中药现代化开辟新世纪。

    Abstract:

    The Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/associated protein 9 (CRISPR /Cas9) immune system is an adaptive immune system widely distributed in bacteria and archaea. It precisely defends against invasion by exogenous phages, viruses, and plasmids through sequence-specific endogenous immune response mechanisms. As the most prominent member of this family, the CRISPR/Cas9 system has evolved into the most widely applied, flexible, and efficient technical platform in the field of genome engineering due to its exceptional genome modification capabilities. Within the CRISPR/Cas9 system, the Cas9 protein, precisely guided by a single-stranded guide RNA (gRNA), can specifically recognize target DNA sequences and induce double-strand breaks. This activates the cell’s DNA repair mechanisms, enabling gene knockout, knock-in, or modification. Demonstrating significant advantages in specificity, flexibility, and operability, CRISPR/Cas9 technology has shown immense potential in the medical field, opening new avenues for modernizing traditional Chinese medicine research. On one hand, this technology can be used to construct precise disease models and tailor personalized treatment plans. It enables in-depth elucidation of the molecular mechanisms underlying the action targets and signaling pathways of traditional Chinese medicine formulas and active components, thereby unraveling the scientific secrets of their complex mechanisms of action. On the other hand, it demonstrates powerful tool value in improving Traditional Chinese Medicine (TCM ) germplasm resources, identifying and screening superior varieties, evaluating the controllability of TCM quality, and producing innovative drugs, providing technical support for the standardization and precision of TCM. Simultaneously, the high-throughput omics data generated by CRISPR technology is driving Artificial Intelligence (AI) to construct virtual disease models and drug prediction systems. This empowers the intelligent screening of effective TCM components, the precise prediction of potential targets, and the exploration of “reducing toxicity while enhancing efficacy” through formula combinations. This synergistic innovation between CRISPR and AI aligns perfectly with precision medicine’s urgent demand for personalized, efficient drug development, injecting new momentum into the modernization and transformation of TCM. This paper first systematically reviews and explains the developmental trajectory, structural basis, and action mechanisms of the CRISPR/Cas9 system, tracing its scientific evolution from a bacterial immune system to a gene-editing tool. It then comprehensively outlines the current state of convergence between precision medicine concepts and modernization research in traditional Chinese medicine, analyzing the synergistic points and potential spaces for their integration. Against the backdrop of rapid precision medicine advancement, this paper emphasizes how CRISPR/Cas9 gene editing technology empowers in-depth analysis of TCM mechanisms—including specific applications in disease model construction, therapeutic target validation, and multi-target network regulation studies. It further elaborates on its multidimensional practical contributions to modernizing TCM, spanning key domains such as germplasm resource innovation, bioactive compound biosynthesis, quality standardization control, and novel TCM drug development. Finally, this paper envisions the future landscape of deep integration between CRISPR technology and AI: from data-driven intelligent drug screening to high-throughput precision discovery of effective TCM components, and further to intelligent model construction based on “reducing toxicity while enhancing efficacy” mechanisms. The synergistic convergence of these multidimensional technologies will pioneer new scientific paradigms and translational pathways for TCM modernization, propelling traditional Chinese medicine toward leapfrogging development in the era of precision medicine.

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王淑贤,郭飞飞,马广强. CRISPR-Cas9技术在中药现代化中的探索[J].生物化学与生物物理进展,,():

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  • 收稿日期:2025-11-13
  • 最后修改日期:2026-03-06
  • 录用日期:2026-03-06
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