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Review Article

探索基因治疗对神经系统疾病的作用

卷 21, 期 1, 2021

发表于: 17 September, 2020

页: [11 - 22] 页: 12

弟呕挨: 10.2174/1566523220999200917114101

价格: $65

摘要

基因治疗是医学突破的前沿领域之一，可以有效地解决以前无法治愈的疾病。将矫正遗传物质或治疗性基因注入细胞可恢复缺失的基因功能，并治愈大量传统医学方法无法治愈的疾病。这一发现有望治疗许多神经退行性疾病，如肌肉萎缩、多发性硬化症、帕金森病(PD)和阿尔茨海默病(AD)等。基因疗法被证明是一种人道的、成本效益高的选择，而不是费力而又及时、不可能的寻找匹配捐赠者和大规模手术的过程。它也克服了传统方法穿越血脑屏障的缺点。然而，只有在对该病的免疫发病机制和分子机制有了深入的了解后，基因治疗的应用才有可能。基因治疗的过程大致可分为三个主要步骤:阐明目的基因，筛选合适的载体，确定最佳的转移方式;每一步都需要进行普及研究。本文就基因治疗在神经退行性疾病中的作用、载体和传递方法进行综述，重点对中枢神经系统(CNS)的基因治疗进行综述。

关键词: 基因治疗，神经退行性疾病，神经病变，中枢神经系统，AAV，载体

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