白血病干细胞是白血病发生、恶化、转移、治疗后复发并产生药物抗性的根源。申请者课题组着眼于从贵州苗药中发现靶向miRNAs而选择性清除白血病干细胞的创新药物，并探讨其分子作用机制。为实现这一目标，申请利用本课题组特有的靶向白血病干细胞更新和分化途径的模型、原代白血病干细胞分离和体外培养技术、人源异殖小鼠模型和白血病小鼠模型，对154种进入国家药品标准的贵州苗药成方制剂进行研究。大量研究表明在白血病干细胞和正常造血细胞表达差异的miRNAs是特异清除白血病干细胞的理想靶标。本项目的目标是利用以上筛选体系，从源于贵州苗药中，发现靶向miRNAs的贵州苗药制剂，并利用这些活性物质探针探讨白血病干细胞中异常信号途径的新基因和新蛋白，同时为开发贵州民族药的新功效和使其走向世界药品市场提供科学理论依据。

Leukemia stem cells play a key role in the progression, metastasis, recurrence and drug resistance of leukemia after treatment. The applicant's research group focused on the formula of Guizhou Miao ethnomedicines that selectively eliminated leukemia stem cells by targeting miRNAs and investigate the molecular mechanism of their action. In order to achieve this goal, using unique model of leukemia stem cell renewal and differentiation pathways, the technologies of leukemia stem cell isolation and culture in vitro, human primary leukemia stem cells xenograft mouse model and the leukemia mouse model, we will investigate 154 species of Guizhou Miao ethnomedicines that meet national drug standard. Numerous studies have shown that differential expression of miRNAs in leukemia stem cells and normal hematopoietic cells are potential targets for the selective elimination of leukemia stem cells. The goal of this project is to use the above screening system to identify Guizhou miao medicine that targeted miRNAs and use the bioactive substance as probes to explore new genes and new proteins in abnormal signaling pathways in leukemia stem cells. At the same time, it provides a scientific basis for the development of the new efficacy of Guizhou Miao ethnomedicines and provide the scientific basis for leading them into the world drug market.