中国药物警戒 ›› 2025, Vol. 22 ›› Issue (9): 975-982.
DOI: 10.19803/j.1672-8629.20250374

• 中药及外泌体临床合理用药探究专栏 • 上一篇    下一篇

单细胞转录组测序技术在中药效/毒机制解析中的研究进展

倪皓雨1,2, 周磊2, 王宁宁2, 杨兴鑫1, 周维2, 沈磐2#, 高月2,3,*   

  1. 1云南中医药大学中药学院,云南 昆明 650500;
    2军事科学院军事医学研究院,北京 100850;
    3中国人民解放军总医院肾脏疾病国家重点实验室,北京 100853
  • 收稿日期:2025-06-16 出版日期:2025-09-15 发布日期:2025-09-22
  • 通讯作者: *高月,女,博士,研究员,中药药理与毒理学。E-mail: gaoyue@bmi.ac.cn。#为共同通信作者。
  • 作者简介:倪皓雨,女,在读硕士,中药毒理学。
  • 基金资助:
    国家自然科学基金资助项目（82304858、82192910、82192911）

Research Advancements in Applications of Single-Cell Transcriptome Sequencing Technology in Mechanism Elucidation of Pharmacodynamics and Toxicity of Traditional Chinese Medicine

NI Haoyu1,2, ZHOU Lei2, WANG Ningning2, YANG Xingxin1, ZHOU Wei2, SHEN Pan2#, GAO Yue2,3,*   

  1. 1Yunnan University of Chinese Medicine, Kunming Yunnan 650500, China;
    2Academy of Military Medical Sciences, Academy of Military Sciences, Beijing 100850, China;
    3State Key Laboratory of Kidney Diseases, Chinese PLA General Hospital, Beijing 100853, China
  • Received:2025-06-16 Online:2025-09-15 Published:2025-09-22

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摘要： 目的 针对当前中药药效和毒性效应难以“说明白、讲清楚”,制约创新研发与临床用药安全的问题,探讨单细胞转录组测序技术（scRNA-seq）在中药研究中的运用情况,为中医药高质量现代化发展提供参考。方法 通过系统梳理当前运用scRNA-seq探究中药效/毒机制的研究进展,重点从疾病治疗领域和毒性特征进行分类阐述,解析scRNA-seq在各项研究中的功能定位与核心发现。结果 归纳scRNA-seq在中药效/毒机制解析中“成分解析-细胞定位-靶点筛选-机制验证”的研究思路范式,发现scRNA-seq能突破中药药效与毒性探究在器官组织层面的宏观观测局限,精确定位至细胞亚群的特异性响应及基因表达动态,可深度阐释中药药物作用与致毒机制的微观本质,并据此提出单细胞网络药理/毒理学的效/毒机制解析策略。结论 scRNA-seq已成为解析中药药理与毒理科学内涵的关键手段之一,围绕该技术构建的中药效/毒研究策略,为推动中药现代化提供参考。

关键词: 中医药, 单细胞转录组测序, 中药药效, 中药毒性, 中药作用机制, 效/毒

Abstract: Objective To explore the applicability of single-cell RNA sequencing (scRNA-seq) technology in TCM research given the current challenges to precise characterization of the therapeutic efficacy and toxicological profiles of Traditional Chinese Medicine (TCM). Methods Recent research advances in utilizing scRNA-seq to investigate the efficacy and toxicity mechanisms of TCM were reviewed in general and related disease treatments and toxicity in particular. The functional positioning and key findings of scRNA-seq in various studies were also analyzed. Results A standardized research paradigm known as "component analysis-cellular localization-target screening-mechanism validation" was established to explore the efficacy and toxicity of TCM using scRNA-seq that could overcome the limitations of traditional organ-level observations and reveal the microscopic essence of TCM’s pharmacological and toxicological actions by enabling precise identification of cellular subpopulation-specific responses and dynamic gene expression changes. Based on these insights, we proposed a single-cell network pharmacological or toxicological strategy for systematic elucidation of mechanisms. Conclusion scRNA-seq has emerged as a pivotal methodology for deciphering what is underlying the pharmacology and toxicology of TCM. Research strategies in TCM using this technology can provide novel perspectives and references for advancing the modernization of TCM.

Key words: Traditional Chinese Medicine (TCM), Single-Cell Transcriptome Sequencing (scRNA-seq), Pharmacodynamics of Traditional Chinese Medicine, Toxicity of Traditional Chinese Medicine, Mechanisms of Action of Traditional Chinese Medicine, Pharmacodynamics and Toxicity

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