Xenotransplantation of patient-derived samples in mouse models has been instrumental in depicting the role of hematopoietic stem and progenitor cells in the establishment as well as progression of hematological malignancies. The foundations for this field of research have been based on the development of immunodeficient mouse models, which provide normal and malignant human hematopoietic cells with a supportive microenvironment. Immunosuppressed and genetically modified mice expressing human growth factors were key milestones in patient-derived xenograft (PDX) models, highlighting the importance of developing humanized microenvironments. The latest major improvement has been the use of human bone marrow (BM) niche–forming cells to generate human–mouse chimeric BM tissues in PDXs, which can shed light on the interactions between human stroma and hematopoietic cells. Here, we summarize the methods used for human hematopoietic cell xenotransplantation and their milestones and review the latest approaches in generating humanized BM tissues in mice to study human normal and malignant hematopoiesis.

在小鼠模型中对源自患者的样本进行异种移植有助于描述造血干细胞和祖细胞在血液系统恶性肿瘤的建立和进展中的作用。该研究领域的基础基于免疫缺陷小鼠模型的开发，该模型为正常和恶性人类造血细胞提供了支持性微环境。表达人类生长因子的免疫抑制和转基因小鼠是患者来源的异种移植 (PDX) 模型中的关键里程碑，突出了开发人性化微环境的重要性。最新的重大改进是使用人骨髓 (BM) 生态位形成细胞在 PDX 中生成人-鼠嵌合 BM 组织，这可以阐明人类基质和造血细胞之间的相互作用。在这里，我们总结了用于人类造血细胞异种移植的方法及其里程碑，并回顾了在小鼠中生成人源化 BM 组织以研究人类正常和恶性造血的最新方法。