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Updated: Jun 12, 2025
A Familial Hypercholesterolemia Human Liver Chimeric Mouse Model Using Induced Pluripotent Stem Cell-derived Hepatocytes
Published on: September 15, 2018
Beyond conventional treatment: ASGR1 Leading the new era of hypercholesterolemia management
Jiali Song1, Yang Fang1, Xiuqin Rao1
1Department of Anesthesiology, the Second Affiliated Hospital of Nanchang University, Nanchang, Jiangxi 330006, PR China; Key Laboratory of Anesthesiology of Jiangxi Province, 1# Minde Road, Nanchang, Jiangxi 330006, PR China.
Insights
Asialoglycoprotein receptor 1 (ASGR1) is key for cholesterol metabolism and low-density lipoprotein (LDL) clearance. Targeting ASGR1 offers a novel approach for managing hypercholesterolemia and reducing cardiovascular disease (CVD) risk.
Area of Science:
- Biochemistry
- Cardiology
- Molecular Biology
Background:
- Cardiovascular disease (CVD) is a leading cause of death globally.
- Hypercholesterolemia is a significant risk factor for CVD.
- Current lipid-lowering therapies are insufficient for many patients.
Purpose of the Study:
- To review the biological functions and regulatory mechanisms of ASGR1 in cholesterol metabolism.
- To explore ASGR1 as a therapeutic target for hypercholesterolemia and related CVDs.
- To analyze ASGR1's role in liver-specific pathways and its association with CVD risk.
Main Methods:
- Literature review of ASGR1 research.
- Analysis of ASGR1's role in lipid homeostasis.
- Examination of ASGR1 variants and their implications in CVD risk.
Main Results:
- ASGR1, primarily on hepatocytes, regulates cholesterol metabolism and LDL clearance.
- ASGR1 plays a crucial role in liver-specific lipid pathways.
- ASGR1 variants are linked to altered CVD risk.
Conclusions:
- ASGR1 is a promising therapeutic target for hypercholesterolemia.
- Understanding ASGR1 mechanisms can lead to novel CVD treatments.
- Further research into ASGR1 is essential for clinical applications.
Abstract:
Cardiovascular disease (CVD) remains a leading cause of mortality worldwide, with hypercholesterolemia being a major risk factor. Although various lipid-lowering therapies exist, many patients fail to achieve optimal cholesterol control, highlighting the need for novel therapeutic approaches. ASGR1 (asialoglycoprotein receptor 1), predominantly expressed on hepatocytes, has emerged as a key regulator of cholesterol metabolism and low-density lipoprotein (LDL) clearance. This receptor's ability to regulate lipid homeostasis positions it as a promising target for therapeutic intervention in hypercholesterolemia and related cardiovascular diseases. This review critically examines the biological functions and regulatory mechanisms of ASGR1 in cholesterol metabolism, with a focus on its potential as a therapeutic target for hypercholesterolemia and related cardiovascular diseases. By analyzing recent advances in ASGR1 research, this article explores its role in liver-specific pathways, the implications of ASGR1 variants in CVD risk, and the prospects for developing ASGR1-targeted therapies. This review aims to provide a foundation for future research and clinical applications in hypercholesterolemia management.
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