摘要
近年来,已经研究了几种靶向基因治疗(GT)的方法。但是,细胞外囊泡(EVs)作为在细胞间传递遗传信息的穿梭物的出现,引起了科学界的极大兴趣。由于它们在处理短序列核酸(mRNA,miRNA),蛋白质,重组蛋白,外来体方面具有更高的能力,因此,最流行的细胞外囊泡形式被视为可靠的生物治疗载体。它们具有通过每个生物膜的天然通道,可用于特定部位和有效的药物输送,而不会引起任何免疫反应,因此,它们是理想的输送媒介。另外,在过去的几十年中,进行了许多研究,利用外泌体介导的基因疗法开发有效的疗法,其概念具有更高的基因分离,纯化和递送机制加载,递送和靶向方案的精确度。这篇综述讨论了有助于开发有效的基因治疗方案的几个方面,重点介绍了与当前GT相关的局限性和缺点以及建议的治疗方案。
关键词: 细胞外囊泡(EVs),病毒载体,基因组,microRNA(miRNA),short hairpin RNA(shRNA),免疫原性。
图形摘要
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