目的观察小干扰RNA(siRNA)对人淀粉样前体蛋白(APP)基因的沉默作用。方法将设计好的一对寡核苷酸在细胞内源性地转录成小发夹RNA(shRNA),同时构建了携带2个APP亚型的增强型绿色荧光蛋白(EGFP)的GFPAPP重组质粒;利用EGFP作为报告基因,在COS7细胞内通过共转染siRNA表达质粒和GFPAPP重组质粒来观察沉默效应。结果针对APP的siRNA可有效地下调APP基因。结论针对APP的siRNA将不仅在研究APP基因功能和深入研究阿尔茨海默病(AD)的病理分子机制上,而且在运用siRNA这种新型的反义核酸药物治疗AD方面扮演重要角色。 Objective To observe the silencing of human amyloid precursor protein (APP) gene by small interfering RNA (siRNA). Methods A designed pair of oligonucleotides was endogenously transcribed into small hairpin RNAs (shRNAs) in cells. At the same time, recombinant GFPAPP plasmids carrying enhanced green fluorescent protein (EGFP) of two APP subtypes were constructed. Using EGFP as a reporter gene, silencing effects were observed in COS7 cells by co-transfecting siRNA expression plasmids and GFPAPP recombinant plasmids. Results siRNA targeting APP effectively down-regulated APP gene. Conclusion siRNA targeting APP will play an important role not only in the study of APP gene function and in the further study of the pathological molecular mechanism of Alzheimer’s disease (AD), but also in the treatment of AD with the new antisense nucleic acid siRNA.