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Review Article

非病毒Episomal载体基因治疗的现状和新兴机遇

卷 23, 期 2, 2023

发表于: 27 October, 2022

页: [135 - 147] 页: 13

弟呕挨: 10.2174/1566523222666221004100858

价格: $65

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摘要

基因疗法在管理广泛的遗传疾病方面非常有益己被证明，目前没有或很少有有效的治疗方法。基因转移载体在基因治疗领域具有非常重要的意义。可以将非病毒的附着载体附着在供体细胞染色体上，而不是将其整合，从而消除病毒和整合载体的副作用。它是一种安全的、最优的基因治疗表达载体，因为它不会引起任何不良反应。但克隆率适中，表达量低，克隆数量少，不适合用于基因治疗。自第一代非病毒附着episomal载体构建以来，人们采取了截断MAR元件、降低CpG基序数量、选择合适的启动子和利用调控元件等各种步骤来调控其表达和稳定性。这增加了非病毒附着载体的转染有效性，同时也使其高水平表达并保持高水平的稳定性。载体是基因治疗中常用的一种基因结构，用于治疗各种系统性疾病。本文综述了非病毒附着载体的各种优化策略的开发进展，以及这些载体在基因治疗中的未来应用。

关键词: 附着载体、非病毒载体、载体优化、基因治疗、帕金森病、毒性、免疫反应、疗效。

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