目的：应用网络药理学和分子对接技术，探讨肝豆扶木汤治疗肝豆状核变性的作用机制。

方法：利用TCMSP、UniProt等数据库查询和筛选与肝豆扶木汤相关的化学成分与其作用靶点；通过GeneCards、OMIM、TTD等数据库进行检索，获得肝豆状核变性的相关疾病靶点；利用R语言4.4.0将药物有效成分的作用靶点和疾病靶点取交集获得肝豆扶木汤治疗肝豆状核变性的潜在靶点，并使用VennDiagram包绘制Venn图进行交集靶点可视化。将网络中各节点信息，导入Cytoscape 3.9.1，构建中药-成分-靶点-疾病网络图。调用clusterProfiler、org.Hs.eg.db等包进行GO功能富集分析和KEGG通路富集分析，采用计算机模拟的分子对接技术对核心化合物和核心靶点进行对接验证，以作为本次网络药理学研究成果的初步验证。

结果：在肝豆扶木汤中可能通过槲皮素、β-谷甾醇、山萘酚、豆甾醇及柚皮素等有效成分，作用于BCL2、RELA、PRKCA、PTGS2等核心靶点，主要富集通路为脂质与动脉粥样硬化、肿瘤坏死因子（tumor necrosis factor，TNF）信号通路、PI3K-Akt 信号通路、乙型肝炎、丙型肝炎、白细胞介素（interleukin，IL）-17 信号通路、低氧诱导因子（hypoxia-inducible factor，HIF）-1信号通路、酒精性脂肪肝、肝细胞癌等。分子对接显示关键成分与靶点具有良好的结合能力。

结论：肝豆扶木汤治疗肝豆状核变性的机制具有多活性成分、多靶点和多通路的特点。

Objective: To investigate the mechanism of Gandou Fumu Decoction in the treatment of hepatolenticular degeneration (HLD, also known as Wilson’s disease) using network pharmacology and molecular docking techniques.

Methods: The chemical constituents of Gandou Fumu Decoction and their corresponding targets were retrieved and screened using databases including TCMSP and UniProt. The intersection between the drug-related targets and disease targets was identified using R (v4.4.0) to determine the potential targets through which Gandou Fumu Decoction may exert therapeutic effects on Wilson’s disease, and the VennDiagram package was employed to visualize the overlapping targets via a Venn diagram. All node information from the network was imported into Cytoscape (v3.9.1) to construct a “traditional Chinese medicine-compound-target-disease” network. Gene Ontology (GO) functional enrichment analysis and Kyoto Encyclopedia of Genes and Genomes (KEGG) pathway enrichment analysis were performed using the “clusterProfiler” and “org. Hs. eg. db” packages. Molecular docking simulations were then conducted between the core compounds and key targets to provide preliminary verification of the network pharmacology findings.

Results: Gandou Fumu Decoction may exert its therapeutic effects on Wilson’s disesase through active constituents such as quercetin, β -sitosterol, kaempferol, stigmasterol, and naringin, acting on key targets including B-cell lymphoma 2 (BCL2), RELA proto-oncogene NF- κB subunit (RELA), protein kinase C alpha (PRKCA), and prostaglandin-endoperoxide synthase 2 (PTGS2). The major enriched pathways include lipid metabolism and atherosclerosis, the tumor necrosis factor (TNF) signaling pathway, the PI3K-Akt signaling pathway, hepatitis B, hepatitis C, the interleukin (IL) -17 signaling pathway, the hypoxiainducible factor (HIF)-1 signaling pathway, alcoholic fatty liver disease, and hepatocellular carcinoma. Molecular docking analysis indicates strong binding affinities between the key components and their corresponding targets.

Conclusion: The therapeutic mechanism of Gandou Fumu Decoction in treating Wilson’s disease is characterized by multiple active components, multiple targets, and multiple pathways.