促Utrophin表达药物的筛选方法的建立与应用

A method for screening Utrophin expression activators from traditional Chinese medicine library

目的:建立一种筛选促Utrophin表达药物的方法。方法:将含有utrophin基因下游增强子DUE和PA启动子以及LacZ报告基因的重组质粒转染小鼠成肌细胞C2C12细胞,以此作为细胞模型,筛选中药材样品库,筛选出能增强Utrophin表达的药物,得到的阳性药物分别给予C2C12细胞和BALB/c小鼠,检测Utrophin表达的变化。结果:得到佛手等3个在细胞和整体水平促进Utrophin表达的候选药物。结论:成功地建立了1种筛选促Utrophin表达药物的方法,为进一步开发杜兴氏肌营养不良症(DMD)治疗药物奠定基础。

Aim：To establish a method for screening Utrophin expression activators from traditional Chinese medicine. Methods： C2C12 myoblasts cells were transfected with recombinant plasmid which containing utrophine enhancer DUE, PA promroter and LacZ reporter gene, for screening the traditional Chinese medicine library to find Utrophin expression activators. Results： Three positive traditional Chinese medicine were obtained through high throughput screening with Utrophin reporter model. BALB/c mice were treated by the extract from the three traditional Chinese medicine. The expressions of Utrophin in tibialis anterior muscle of these mice were upregulated and assessed by immunological blot assay. Conclusion： Gradients from the three traditional Chinese medicine stimulate Utrophin expression and may be the candidate drug for Duchenne muscular dystrophy therapy.