Gene therapy is a therapeutic strategy of delivering foreign genetic material (encoding for an important protein) into a patient’s target cell to replace a defective gene. Nucleic acids are embedded within the adeno-associated virus (AAVs) vectors; however, preexisting immunity to AAVs remains a significant concern that impairs their clinical application. Extracellular vesicles (EVs) hold great potential for therapeutic applications as vectors of nucleic acids due to their endogenous intercellular communication functions through their cargo delivery, including lipids and proteins. So far, small RNAs (siRNA and micro (mi)RNA) have been mainly loaded into EVs to treat several diseases, but the potential use of EVs to load and deliver exogenous plasmid DNA has not been thoroughly described. This review provides a comprehensive overview of the principal methodologies currently employed to load foreign genetic material into EVs, highlighting the need to find the most effective strategies for their successful clinical translations.

中文翻译：

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使用负载外源核酸的细胞外囊泡开发新策略。


基因治疗是一种将外源遗传物质（编码重要蛋白质）传递到患者靶细胞中以替换有缺陷基因的治疗策略。核酸嵌入腺相关病毒 (AAV) 载体中；然而，预先存在的对 AAV 的免疫力仍然是一个重大问题，会损害其临床应用。细胞外囊泡（EV）作为核酸载体在治疗应用方面具有巨大的潜力，因为它们通过其货物输送（包括脂质和蛋白质）具有内源性细胞间通讯功能。迄今为止，小RNA（siRNA和微（mi）RNA）主要被加载到EV中以治疗多种疾病，但EV加载和递送外源质粒DNA的潜在用途尚未得到充分描述。这篇综述全面概述了目前用于将外源遗传物质加载到 EV 中的主要方法，强调需要找到最有效的策略来实现其成功的临床转化。