【摘要】 阿尔茨海默病是一种起病隐匿的神经退行性疾病，现有的治疗药物仅能延缓疾病进展，缓解症状。小干扰RNA通过切割编码靶基因的mRNA,显示出治疗阿尔茨海默病的潜力，但将小干扰RNA稳定递送至病灶需要克服诸多挑战。本文描述了阿尔茨海默病的病理机制、现有药物靶点、小干扰RNA疗法的优点与挑战、跨血脑屏障递送系统的研究进展、新型小干扰RNA递送系统和正在进行的阿尔茨海默病药物临床试验等，力求为用于阿尔茨海默病的小干扰RNA疗法的相关研究提供借鉴和参考。更多还原

【Abstract】 Alzheimer’s disease is a neurodegenerative disease with insidious onset, and existing therapeutic drugs can only delay the progression of the disease and relieve symptoms. Small interfering RNAs have shown the potential to treat Alzheimer’s disease by cutting mRNA encoding target genes, but stable delivery of siRNA to lesions requires overcoming many challenges. This review describes the pathological mechanisms of Alzheimer’s disease, existing drug targets, advantages and challenges of siRNA therapy, research advances in delivery systems across the blood-brain barrier, novel siRNA delivery systems, and ongoing clinical trials of Alzheimer’s drugs. We aim to provide reference for the research of siRNA therapy for Alzheimer’s disease.更多还原