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Review Article

基因治疗，一种用于神经疾病的新型治疗工具：当前进展，挑战和未来前景

卷 20, 期 3, 2020

页: [184 - 194] 页: 11

弟呕挨: 10.2174/1566523220999200716111502

价格: $65

摘要

神经系统疾病给医疗保健系统带来了重大威胁，因为它们给社会经济带来了沉重负担。所有老年人群都容易出现一种或其他神经系统疾病，并伴有神经发炎，神经退行性变和认知功能障碍的症状。目前，可用的药物疗法不足以治疗这些疾病，并且在大多数情况下，它们仅提供姑息作用。还发现神经系统疾病的分子病因与遗传组成的改变直接相关，遗传改变可以由伤害，环境毒素和某些现有疾病遗传或触发。因此，为了照顾这种情况，基因治疗已经成为一种先进的方法，声称可以通过缺失，沉默或编辑缺陷基因并通过插入更健康的基因来永久治愈疾病。在这种方式中，载体（病毒的和非病毒的）用于通过各种途径将靶向基因传递到大脑的特定区域。目前，基因治疗已在复杂的神经系统疾病，例如帕金森氏病，阿尔茨海默氏病，亨廷顿病，多发性硬化症，肌萎缩性侧索硬化症和溶酶体贮积病中显示出积极的成果。但是，存在一些局限性，例如免疫原性反应，病毒载体的非特异性以及缺乏有效的生物标记物来了解治疗效果。在改善载体设计，基因选择和靶向递送方面已经取得了相当大的进展。这篇综述文章探讨了神经疾病中基因治疗的现状及其临床相关性，挑战和未来前景。

关键词: CRISPR / Cas9，载体，siRNA，基因编辑，神经修复，临床试验。

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