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Review Article

利用基因治疗载体靶向递送神经退行性疾病:基因下一个治疗目标

卷 21, 期 1, 2021

发表于: 17 August, 2020

页: [23 - 42] 页: 20

弟呕挨: 10.2174/1566523220999200817164907

价格: $65

摘要

基因治疗技术，自从它在近50年前出现以来，一直被科学家用作各种疾病的潜在治疗选择。综述讨论了一些主要的神经退行性疾病(NDDs)像阿尔茨海默病(AD)、帕金森病(PD),运动神经元病(MND),脊髓性肌萎缩(SMA),亨廷顿氏病(HD),多发性硬化症(MS)等和其内在的遗传机制以及基因疗法的作用发挥在打击他们。本文还详细讨论了每一种NDDs的发病机制和基因表达改变的分子机制。基因治疗载体的使用将被证明是治疗性现代医学领域的有效工具。因此，对其实施的持续努力和不断的研究可以为我们提供迄今被认为无法治愈的疾病的强有力的治疗选择。

关键词: 基因治疗，神经退行性疾病，病毒载体，非病毒载体，阿尔茨海默病，帕金森病，精神分裂症逆转录病毒

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