Therapeutic efficacy and multitarget mechanisms of traditional Chinese medicine in hereditary liver diseases: Insights into bioactive components

Table 2 Summary of disease-specific preclinical and clinical evidence for bioactive components of traditional Chinese medicine

Component	Model	Main outcomes	Lever of evidence	Ref.
Berberine	In vitro (cell experiment)	Enhances clearance of aberrant proteins	Preclinical	Rusmini et al[74], 2020
		Directly chelates excess copper and iron ions	Preclinical	Purwaningsih et al[16], 2023
		Inhibits inflammatory response induced by LPS through NF-κB pathway	Preclinical	Reddi et al[93], 2021
		Inhibits ferroptosis	Preclinical	Wang et al[84], 2024
		Indirectly blocks the maturation of IL-1β/IL-18 and the cleavage of GSDMD	Preclinical	El Gazzar et al[92], 2025
	In vivo (animal experiment)	Attenuates fructose-induced insulin resistance in mice	Preclinical	Li et al[111], 2020
		Protects against diabetic retinopathy in mice	Preclinical	Zhai et al[136], 2020
		Significantly improves the composition of intestinal microbiota, increases beneficial bacteria, and inhibits opportunistic pathogens	Preclinical	Dehau et al[126], 2023
		Inhibits osteosarcoma	Preclinical
	Clinical trial/meta-analysis	Efficacy and safety for premature ventricular contractions	Metaanalysis of randomized controlled trials (level 1a evidence)	Qiao et al[34], 2023
Matrine	In vitro (cell experiment)	Indirectly blocks the maturation of IL-1β/IL-18 and the cleavage of GSDMD	Preclinical	Sun et al[91], 2025
		Suppresses the production of core pro-inflammatory cytokines (TNF-α, IL-6)	Preclinical	Sun et al[94], 2019
	In vivo (animal experiment)	Inhibits IL-1β secretion in primary porcine alveolar macrophages	Preclinical	Sun et al[94], 2019
		Potential for multi-targeted intervention in hereditary liver diseases	Preclinical	Sun et al[33], 2022
	Clinical trial/meta-analysis	No specific clinical trial or meta-analysis evidence for hereditary liver diseases mentioned in the provided text
Quercetin	In vitro (cell experiment)	Directly scavenges free radicals	Preclinical	Afanas'ev et al[65], 1989
		Directly interacts with misfolded proteins to inhibit their aggregation	Preclinical	Alghamdi et al[75], 2022
		Protects ethanol-induced hepatocyte pyroptosis	Preclinical	Zhao et al[123], 2022
		Acts as a potent antioxidant	Preclinical	Aghababaei and Hadidi[79], 2023
		Suppresses the production of core pro-inflammatory cytokines (TNF-α, IL-6)	Preclinical	Li et al[95], 2021
		As a direct antioxidant	Preclinical	Remigante et al[110], 2022
	In vivo (animal experiment)	Attenuates the activation of hepatic stellate cells and liver fibrosis in mice	Preclinical	Li et al[132], 2016
		Inhibits hepatic gluconeogenesis in diabetic rats	Preclinical	Chen et al[115], 2020
		Modulates T-cell homeostasis	Preclinical	Ke et al[105], 2023
		Regulates the expression of genes associated with hepatic lipid metabolism	Preclinical	Luo et al[155], 2025
		Antidiabetic effects in diabetic rats (in vivo and in silico studies)	Preclinical	Abdou et al[114], 2025
		Fabrication and in vitro/vivo evaluation of quercetin nanocrystals for liver targeted drug delivery	Preclinical	Shen et al[38], 2024
	Clinical trial/meta-analysis	No specific clinical trial or meta-analysis evidence for hereditary liver diseases mentioned in the provided text
Baicalin	In vitro (cell experiment)	Directly scavenges free radicals	Preclinical	Liang et al[66], 2009
		Interferes with the IRE1α/TRAF2/JNK pathway	Preclinical	Hao et al[69], 2021
		Acts as a potent antioxidant	Preclinical	Zhao et al[82], 2020
		Indirectly blocks the maturation of IL-1β/IL-18 and the cleavage of GSDMD	Preclinical	Rui et al[90], 2020; Song et al[102], 2025
		Suppresses the production of core pro-inflammatory cytokines (TNF-α, IL-6)	Preclinical	He et al[96], 2022
		Inhibits HSC proliferation, apoptosis, invasion, migration, and activation	Preclinical	Wu et al[138], 2018
	In vivo (animal experiment)	Ameliorates experimental liver cholestasis in mice	Preclinical	Shen et al[41], 2017
		Inhibited CYP7A1 expression, improved bile acid, and glycolipid metabolism in T2DM mice	Preclinical	Yan et al[131], 2022
		Ameliorates atherosclerosis in apolipoprotein E-deficient mice	Preclinical	Zhao et al[82], 2020
	Clinical trial/meta-analysis	No specific clinical trial or meta-analysis evidence for hereditary liver diseases mentioned in the provided text
Ursolic acid	In vitro (cell experiment)	Enhances the expression of endogenous antioxidant enzyme systems	Preclinical	Fu et al[45], 2023
		Exerts ferroptosis-defensive effects	Preclinical	Fu et al[45], 2023
	In vivo (animal experiment)	Reduces oxidative stress injury to ameliorate experimental autoimmune myocarditis in mice	Preclinical	Fu et al[45], 2023
		Ameliorates hepatic steatosis and improves metabolic disorders in high-fat diet-induced non-alcoholic fatty liver disease rats	Preclinical	Li et al[118], 2014
		Activates Nrf2, an endogenous defense hub	Preclinical	Wang et al[106], 2023; Bak et al[108], 2017
		Suppresses TGF-β1-induced quiescent HSC activation and transformation	Preclinical	Yu et al[140], 2017
	Clinical trial/meta-analysis	No specific clinical trial or meta-analysis evidence for hereditary liver diseases mentioned in the provided text
Oleanolic acid	In vitro (cell experiment)	Exerts ferroptosis-defensive effects	Preclinical	Wang et al[87], 2025
	In vivo (animal experiment)	Alleviates TNBS-induced ulcerative colitis in rat	Preclinical	Wang et al[87], 2025
		Hepatoprotection	Preclinical	Wang et al[46], 2018
		Activates the peroxisome proliferator-activated receptor α signaling pathway	Preclinical	Wang et al[46], 2018
		Activates Nrf2, an endogenous defense hub	Preclinical	Bojuan et al[107], 2025; Bak et al[108], 2017
		Attenuates activation of hepatic stellate cells	Preclinical	Leilei et al[141], 2022
	Clinical trial/meta-analysis	No specific clinical trial or meta-analysis evidence for hereditary liver diseases mentioned in the provided text
Astragalus polysaccharide	In vitro (cell experiment)	Alleviates ERS	Preclinical	Wang et al[71], 2009
		Enhances the expression of endogenous antioxidant enzyme systems	Preclinical	Sha et al[64], 2023
		Inhibits the activation of the NLRP3 inflammasome	Preclinical	Tian et al[98], 2017
	In vivo (animal experiment)	Upregulates hepcidin expression and reduces iron overload in mice	Preclinical	Ren et al[48], 2016
		Improves insulin sensitivity in 3T3-L1 adipocytes	Preclinical	Zhang et al[112], 2018
		Mitigates transport stress-induced hepatic metabolic stress in chicks	Preclinical	Zhao et al[125], 2022
		Significantly improves the composition of intestinal microbiota, increases beneficial bacteria, and inhibits opportunistic pathogens	Preclinical	Zhao et al[127], 2023
		Attenuates murine colitis	Preclinical	Tian et al[98], 2017
	Clinical trial/meta-analysis	No specific clinical trial or meta-analysis evidence for hereditary liver diseases mentioned in the provided text
Ganoderma lucidum polysaccharide	In vitro (cell experiment)	May exert effects through direct or indirect mechanisms to inhibit misfolded protein aggregation	Preclinical	Zeng et al[76], 2017
	In vivo (animal experiment)	Improves rat DSS-induced colitis	Preclinical	Xie et al[128], 2019
		Anti-inflammatory and hepatoprotective effects against carbon tetrachloride-induced liver injury in Kunming Mice	Preclinical	Chen et al[99], 2019
		Inhibits the activation of the NLRP3 inflammasome	Preclinical	Chen et al[99], 2019
		Indirectly maintains metabolic homeostasis	Preclinical	Pan et al[124], 2021
	Clinical trial/meta-analysis	No specific clinical trial or meta-analysis evidence for hereditary liver diseases mentioned in the provided text
Ginsenoside Rg1	In vitro (cell experiment)	Interferes with the IRE1α/TRAF2/JNK pathway	Preclinical	Li et al[70], 2017
		Acts as a potent antioxidant	Preclinical	Li et al[83], 2021
	In vivo (animal experiment)	Protects cardiomyocytes against hypoxia/reoxygenation injury	Preclinical	Li et al[70], 2017
		Inhibits dietary-induced obesity and improves obesity-related glucose metabolic disorders	Preclinical	Li et al[113], 2018
		Inhibits inflammatory responses in alcoholic hepatitis	Preclinical	Li et al[134], 2018
		Ameliorates cardiac oxidative stress and inflammation in streptozotocin-induced diabetic rats	Preclinical	Qin et al[121], 2019
		Ameliorates aging-induced liver fibrosis in SAMP8 mice	Preclinical	Li et al[83], 2021
		Regulates the polarization of macrophages from M1 to M2 phenotype	Preclinical	Zhen et al[103], 2024
	Clinical trial/meta-analysis	No specific clinical trial or meta-analysis evidence for hereditary liver diseases mentioned in the provided text
Saikosaponin	In vitro (cell experiment)	No specific in vitro evidence for hereditary liver diseases mentioned in the provided text	Preclinical
	In vivo (animal experiment)	Inhibits the activation of the NLRP3 inflammasome	Preclinical	Lin et al[100], 2018
		Inhibits peritoneal fibrosis in rats with renal failure	Preclinical	Ruiqi et al[142], 2021
	Clinical trial/meta-analysis	No specific clinical trial or meta-analysis evidence for hereditary liver diseases mentioned in the provided text
Salvianolic acid B	In vitro (cell experiment)	Directly chelates excess copper and iron ions	Preclinical	Xie et al[17], 2025
		Modulates the PERK/eIF2α/CHOP signaling axis	Preclinical	Mai et al[68], 2020
		Acts as a potent antioxidant	Preclinical	Wu et al[81], 2009
		As a direct antioxidant	Preclinical	Xiao et al[109], 2020
	In vivo (animal experiment)	Protects against acute and chronic liver injury	Preclinical	Tao et al[133], 2021
		Protects the integrity of mitochondrial membrane potential	Preclinical	Zheng et al[122], 2020
	Clinical trial/meta-analysis	No specific clinical trial or meta-analysis evidence for hereditary liver diseases mentioned in the provided text
Resveratrol	In vitro (cell experiment)	Modulates the PERK/eIF2α/CHOP signaling axis	Preclinical	Liu et al[67], 2014
		Upregulates the expression of glutathione peroxidase 4	Preclinical	Ni et al[86], 2023
		Acts as a potent antioxidant	Preclinical	Liu et al[80], 2022
		Regulates lipid metabolism in hepatocytes	Preclinical	Wang et al[117], 2024
	In vivo (animal experiment)	Attenuates myocardial injury	Preclinical	Liu et al[80], 2022
		Accelerates wound healing in diabetic mice	Preclinical	Ding et al[104], 2022
		Enhances mitochondrial biogenesis and improves ATP production efficiency	Preclinical	Zhang et al[62], 2017
		Alleviates intestinal mucosal barrier dysfunction in dextran sulfate sodium-induced colitis mice	Preclinical	Pan et al[130], 2020
		Synergistic antitumorigenic activity with calcitriol in triple negative breast cancer xenografts	Mediated by angiogenesis inhibition	García-Quiroz et al[150], 2019
	Clinical trial/meta-analysis	No specific clinical trial or meta-analysis evidence for hereditary liver diseases mentioned in the provided text
Curcumin	In vitro (cell experiment)	Enhances the clearance of aberrant proteins	Preclinical	Liu et al[72], 2023; Zhang et al[73], 2016
		Effectively inhibits the activation of the NLRP3 inflammasome	Preclinical	Sun et al[89], 2017
		Acts as a potent antioxidant	Preclinical	Xiong et al[78], 2025
		Upregulates the expression of glutathione peroxidase 4	Preclinical	Yuan et al[85], 2023
		Suppresses the production of core pro-inflammatory cytokines (TNF-α, IL-6)	Preclinical	Kong et al[97], 2025
		Inhibits the activation of the NLRP3 inflammasome	Preclinical	Kong et al[101], 2016
		Significantly reduces lipid synthesis and accumulation in the liver	Preclinical	Tung et al[116], 2017
	In vivo (animal experiment)	Restrains oxidative stress after intracerebral hemorrhage in rat	Preclinical	Duan et al[63], 2022
		Protects against the intestinal ischemia-reperfusion injury	Preclinical	Tian et al[129], 2016
		Downregulates the expression of TGF-β1	Preclinical	Song et al[139], 2011
		Synergistic hepatoprotective activity with rosemary essential oil	Preclinical	Mahmoudi et al[151], 2022
		Comparison of the efficacy of curcumin and its nano formulation on dexamethasone-induced hepatic steatosis, dyslipidemia, and hyperglycemia in Wistar rats	Preclinical	Hamed et al[159], 2024
	Clinical trial/meta-analysis	No specific clinical trial or meta-analysis evidence for hereditary liver diseases mentioned in the provided text

LPS: Lipopolysaccharide; NF-κB: Nuclear factor-κB; IL: Interleukin; GSDMD: Gasdermin D; TNF-α: Tumor necrosis factor-α; HSC: Hepatic stellate cell; Nrf2: Nuclear factor erythroid 2-related factor 2; TGF-β1: Transforming growth factor β1; ERS: Endoplasmic reticulum stress; NLRP3: NOD-, LRR- and pyrin domain-containing protein 3; DSS: Dextran sulfate sodium.