纳米医学结合靶向基因治疗阿尔茨海默病的现状、挑战与未来  

Nanomedicine plus targeted gene therapy for Alzheimer’s disease:current situation,challenges,and future

作　　者：梁文丹琪 郝梦琪 古源楷 卢欣宇 季文博 庄建华 尹又 LIANG Wendanqi;HAO Mengqi;GU Yuankai;LU Xinyu;JI Wenbo;ZHUANG Jianhua;YIN You(School of Health Science and Engineering,University of Shanghai for Science and Technology,Shanghai 200003,China;Department of Neurology,The Second Affiliated Hospital of Naval Medical University(Second Military Medical University),Shanghai 200003,China)

机构地区：[1]上海理工大学健康科学与工程学院,上海200003 [2]海军军医大学(第二军医大学)第二附属医院神经内科,上海200003

出　　处：《海军军医大学学报》2024年第6期748-755,共8页Academic Journal of Naval Medical University

基　　金：上海市自然科学基金(22ZR147750);海军军医大学(第二军医大学)第二附属医院创新型临床研究项目(2020YLCYJ-Y02).

摘　　要：阿尔茨海默病(AD)是一种神经退行性疾病,以认知、情感、语言和记忆的进行性损害为主要特征。现有的AD治疗手段主要是药物治疗,但药物治疗仅能缓解其症状,并不能有效延缓病情进展。因此,探索有效治疗AD的手段至关重要。研究证实,基因突变和家族遗传与AD的发生、发展密切相关,且基因治疗在AD动物模型中已有所进展。但普通药物与一些小分子药物常因无法穿越血脑屏障难以在脑内发挥作用,而纳米载体则能通过免疫逃逸等方法解决这一问题。本文综述与AD相关的基因治疗策略,总结现有的基因疗法与纳米医学相结合的AD治疗理论,为AD的临床治疗提供新思路。Alzheimer’s disease(AD)is a neurodegenerative disorder characterized by progressive impairment of cognition,emotion,language,and memory.Existing treatments for AD are mainly pharmacological treatments,but they can only improve patients’clinical symptoms and cannot effectively slow down the progression of the disease.Therefore,finding effective treatments for AD is urgent.Research has confirmed that gene mutation and family inheritance are closely related to the development and progression of AD,and gene therapy has made some progress in animal models of AD.Common drugs and some small molecule drugs are often unable to enter the brain through the blood-brain barrier,while nanocarriers can solve this problem through immune escape or other methods.In this paper,we review the gene therapy strategies related to AD and summarize the existing gene therapy and nanomedicine combination of AD therapeutic theories,so as to provide new ideas for the clinical treatment of AD.

关 键 词：阿尔茨海默病 基因治疗 纳米医学 靶向治疗 

分 类 号：R749.16[医药卫生—神经病学与精神病学]