脂肪肝、肝硬化、肝癌和肝衰竭等肝脏疾病的高发病率和高复杂性对传统的研究模型提出了严峻挑战，而肝脏类器官作为一种新型三维（3D）体外模型，具有自主更新、分化和肝组织高度相似的特点，能够高度模拟体内肝组织的生理结构和功能，已成为肝脏疾病研究、药物研发、毒性评估等的重要工具。聚焦于肝脏类器官的研究进展和应用，介绍肝脏类器官的定义、分类和核心优势，梳理肝脏类器官在生理功能模拟、疾病模型构建、药物毒性评估、精准医疗以及再生医学方面的应用进展，并且对当前肝脏类器官研究所面临的血管化不足、标准化缺失、监管与伦理规范不完善等问题进行深入分析，旨在为肝脏类器官相关研究及其在非临床与临床应用的转化提供参考和指引。

The high incidence and complexity of liver diseases, including fatty liver disease, cirrhosis, hepatocellular carcinoma, and liver failure, pose significant challenges to traditional research models. As a novel three-dimensional (3D) in vitro model, liver organoids exhibit self-renewal and differentiation capabilities, closely mirroring the physiological structure and function of human liver tissue. They have emerged as an important tool for studying liver diseases, advancing drug development, and conducting toxicity assessments. This review focuses on recent progress and applications of liver organoids, introducing their definition, classification, and core advantages. It outlines advances in their use for simulating physiological functions, constructing disease models, evaluating drug toxicity, enabling precision medicine, and contributing to regenerative medicine. The article also provides an in-depth analysis of existing challenges, such as insufficient vascularization, lack of standardization, and incomplete regulatory and ethical frameworks. The aim is to offer reference and guidance for future research on liver organoids and to facilitate their translation into non-clinical and clinical applications.

R965.2

药品监管科学全国重点实验室课题（2025SKLDRS0325）