人源化小鼠模型的发展及其在血液学研究中的应用  

The Development of Humanized Mouse Models and Their Applications in Hematological Research

作　　者：陆路 张栩苗 宋远斌 LU Lu;ZHANG Xumiao;SONG Yuanbin(State Key Laboratory of Oncology in South China,Guangdong Provincial Clinical Research Center for Cancer,Sun Yat-sen University Cancer Center,Guangzhou 510060,China)

机构地区：[1]华南恶性肿瘤防治全国重点实验室,广东省恶性肿瘤临床医学研究中心,中山大学肿瘤防治中心,广州510060

出　　处：《中国细胞生物学学报》2025年第3期486-495,共10页Chinese Journal of Cell Biology

基　　金：国家自然科学基金(批准号:82170137);国家自然科学基金优秀青年科学基金项目(海外)(批准号:21HAA01903);广东省自然科学基金面上项目(批准号:2024A1515013182);中央高校基本科研业务费用、中山大学基本科研业务专项(批准号:22hytd13);中山大学百人计划人才基金(批准号:2021012);中山大学肿瘤防治中心高层次人才特殊支持计划(批准号:YTP-SYSUCC-0052)资助的课题。

摘　　要：人源化小鼠作为临床前动物模型,跨越了人、鼠的物种差异和免疫排斥特性,可深入探索疾病的发病机理和药物作用机制,在疾病机制研究以及药物评价中具有重要作用。人、鼠在造血发育方面存在相似性,但在特定谱系的生成和发育上存在显著差异,其造血分子调控机制及模式也不尽相同。近年来,由于基因编辑及异种移植等技术的不断革新,人源化小鼠在血液系统疾病研究中取得了长足发展,该综述涵盖了人、鼠造血发育差异的探讨,人源化小鼠建立发展的过程以及在血液系统疾病中的应用进展。As a preclinical animal model,humanized mice transcend species differences and immune rejection characteristics between humans and mice,and can deeply explore the pathogenesis and drug action mechanisms of diseases.They play an important role in disease mechanism research and drug evaluation.There are similarities in hematopoietic development between humans and mice,but significant differences exist in the generation and development of specific lineages,and their hematopoietic molecular regulatory mechanisms and patterns are also different.In recent years,due to the continuous innovation of gene editing and xenotransplantation technologies,humanized mice have made significant progress in the study of hematological diseases.This review covers the differences in hematopoietic development between humans and mice,the process of establishing and developing humanized mice,and the application progress in hematological diseases.

关 键 词：人源化小鼠 造血干细胞 免疫重建 造血发育 异常造血 

分 类 号：R28[医药卫生—中药学]