Chen Shun, Ge Xuemei, Chen Yinghui, Lv Nan, Liu Zhenguo, Yuan Weien
Department of Neurology, Xinhua Hospital affiliated to Shanghai Jiaotong University School of Medicine, Shanghai Jiaotong University, Shanghai.
Drug Des Devel Ther. 2013;7:117-25. doi: 10.2147/DDDT.S40229. Epub 2013 Feb 22.
Alzheimer's disease (AD) is a progressive neurodegenerative disorder characterized clinically by memory and cognitive dysfunction. Unfortunately, there is no effective therapeutic method for AD treatment or ways to halt disease progression. Many mechanisms are involved in the disease, including genes mutation and protein dysfunction. RNA interference (RNAi) technology may potentially be able to control AD. It can inhibit the protein expression of specific genes by activating a sequence-specific RNA degradation process. This is a powerful tool with which to study gene function, investigate the mechanism of the disease, and validate drug targets. In this review, we highlight the advances in RNAi technology in the investigation and treatment of AD.
阿尔茨海默病（AD）是一种进行性神经退行性疾病，临床上以记忆和认知功能障碍为特征。不幸的是，目前尚无有效的治疗方法来治疗AD或阻止疾病进展。该疾病涉及多种机制，包括基因突变和蛋白质功能障碍。RNA干扰（RNAi）技术可能有潜力控制AD。它可以通过激活序列特异性RNA降解过程来抑制特定基因的蛋白质表达。这是一种用于研究基因功能、探究疾病机制和验证药物靶点的强大工具。在本综述中，我们重点介绍了RNAi技术在AD研究和治疗方面的进展。
