肌萎缩侧索硬化基因治疗研究进展  

Research progress in gene therapy for amyotrophic lateral sclerosis

作　　者：马志 赵慧慧 牛琦 Ma Zhi;Zhao Huihui;Niu Qi(Department of Geriatric Neurology,the First Affiliated Hospital of Nanjing Medical University,Nanjing 210029,China)

机构地区：[1]南京医科大学第一附属医院老年神经内科,南京210029

出　　处：《中华神经科杂志》2022年第3期260-265,共6页Chinese Journal of Neurology

基　　金：国家自然科学基金 (82071434);江苏省自然科学基金 (BK20201490)。

摘　　要：肌萎缩侧索硬化是由大脑和脊髓运动神经元丢失引起的神经退行性疾病,目前尚无有效的治愈方法。基因疗法可以通过传递转基因取代或纠正有缺陷基因,也可以传递神经营养因子的表达,为肌萎缩侧索硬化的治疗带来了希望。基因治疗载体包括病毒载体和非病毒载体。慢病毒载体能够传递治疗序列至中枢神经系统的运动神经元,腺病毒相关病毒也可以有效地介导基因表达和神经营养因子的传递。此外,基因编辑和反义寡核苷酸疗法也是有希望的治疗方案。文中从基础实验和临床试验的角度概述了肌萎缩侧索硬化基因疗法的临床前景。Amyotrophic lateral sclerosis is a neurodegenerative disease caused by the loss of motor neurons in the brain and spinal cord.There is currently no effective cure.The emergence of gene therapy brings hope to treatment,which can be achieved by delivering transgenes to replace or correct defective genes,as well as the expression of neurotrophic factors.The vectors of gene therapy can be viral vectors and non-viral vectors.Lentiviral vectors can be used to deliver therapeutic sequences to motor neurons in the central nervous system.Adeno-associated viruses can effectively mediate gene expression and delivery of neurotrophic factors.Gene editing and antisense oligonucleotides therapy are also perspective treatment options.This article summarizes gene therapy for amyotrophic lateral sclerosis from basic experiments and clinical trials.

关 键 词：肌萎缩侧索硬化 寡核苷酸类 反义 基因治疗 腺病毒相关病毒 

分 类 号：R744.8[医药卫生—神经病学与精神病学] R450[医药卫生—临床医学]