目前，造血干细胞的体外扩增及体内再生用于骨髓型放射病的有效治疗仍存在一些瓶颈问题有待解决。干细胞因子SCF在造血系统中具有至关重要的作用，基于SCF/c-kit轴筛选并获得新型小分子，有望用于体外扩增造血干细胞或成为骨髓型放射病治疗的候选药物。我们拟基于SCF/c-kit靶标，利用小分子化合物库及高内涵成像分析系统筛选的策略筛选并获得可促进基质细胞产生SCF蛋白或激活c-kit受体的小分子，通过体外实验验证筛选到的小分子是否能调节SCF/c-kit。我们已经开展筛选工作，并筛选到3个可刺激SCF生成的小分子化合物，将进一步利用细胞模型筛选并评价筛选到的小分子是否能促进人造血干细胞发育及造血干/祖细胞扩增；且通过体内实验评价筛到的小分子对骨髓辐射损伤的预防或治疗作用，明确筛到的化合物作用的分子机制。研究结果将为我们今后开发出造血干细胞扩增的新方法及治疗骨髓辐射损伤的新型药物奠定基础。

To date, there is still no effective therapeutic drug for radiation-induced bone marrow damage. Stem cell factor (SCF) plays important role in hematopoiesis and hematipoietic stem cell regeneration.Obtaining small molecules that target to SCF or its receptor c-kit might function on hematopoietic system and show application potential. We plan to use high content screening strategy basing on immunostaining against SCF or p-c-kit to search new small molecules that can enhance SCF expression or activate c-kit receptor.We will evaluate the function of these small molecules on promoting hematopoietic stem cell development and expansion. We also plan to use the lethally-irradiated mice to evaluate the effect of these small molecules on the repair of damaged hematopoietic system. In combination with in vitro and in vivo experiments, we will investigate the regulatory mechanism, the downstream signal pathway and the target genes of the small molecules. The project will provide a novel strategy to find a therapeutic molecule via regulation of hematopoietic stem/progenitor cells or their niche.