基因治疗与阿尔茨海默病  

Gene therapy and Alzheimer's disease

作　　者：李建[1,2] 李闻文[1] 周军[2] 

机构地区：[1]中南大学湘雅医学院医学检验系,长沙410013 [2]中南大学湘雅医院医学科学研究中心,长沙410008

出　　处：《中南大学学报（医学版）》2015年第4期428-432,共5页Journal of Central South University ：Medical Science

基　　金：湖南省科技厅国际合作项目(2011WK3047)~~

摘　　要：阿尔茨海默病(Alzheimer’s disease,AD)是一种进行性的神经退行性疾病,临床上表现为记忆和认知功能减退、行为异常和语言功能障碍。其病理特点表现为神经元外β-淀粉样蛋白聚集形成老年斑、细胞内异常磷酸化的tau蛋白聚集形成神经纤维缠结、神经元的缺失和脑血管淀粉样变性。目前用于AD临床治疗的药物有乙酰胆碱酯酶抑制剂、NMDA受体拮抗剂、非甾体抗炎药等,这些药物仅能缓解AD的症状,而基因治疗通过载体介导的转基因技术可以将DNA或RNA直接导入靶细胞,实现保护性或治疗性蛋白的表达及某些致病基因的沉默,从根本上治疗AD。Alzheimer ’s disease(AD) is a progressive neurodegenerative disease characterized by the presence of extracellular β-amyloid in the senile plaques, intracellular aggregates of abnormal phosphorylation of tau protein in the neurofibrillary tangles, neuronal loss and cerebrovascular amyloidosis. The manifestations of clinical symptoms include memory impairment, cognitive decline, altered behavior and language deficit. Currently available drugs in AD therapy consist of acetylcholinesterase inhibitors, NMDA receptor antagonists, non-steroidal anti-inflammatory drugs, etc. Th ese drugs can only alleviate the symptoms of AD. Gene therapy is achieved by vectormediated gene transfer technology, which can delivery DNA or RNA into target cells to promote the expression of a protective or therapeutic protein and silence certain virulence genes.

关 键 词：阿尔茨海默病 基因治疗 载体 策略 

分 类 号：R749.16[医药卫生—神经病学与精神病学] R450[医药卫生—临床医学]