Small interfering RNA (siRNA) is a powerful tool for gene silencing that has been used for a wide range of biomedical applications, but there are many challenges facing its therapeutic use in vivo. Here, we report on a platelet cell membrane-coated metal-organic framework (MOF) nanodelivery platform for the targeted delivery of siRNA in vivo. The MOF core is capable of high loading yields, and its pH sensitivity enables endosomal disruption upon cellular uptake. The cell membrane coating provides a natural means of biointerfacing with disease substrates. It is shown that high silencing efficiency can be achieved in vitro against multiple target genes. Using a murine xenograft model, significant antitumor targeting and therapeutic efficacy are observed. Overall, the biomimetic nanodelivery system presented here provides an effective means of achieving gene silencing in vivo and could be used to expand the applicability of siRNA across a range of disease-relevant applications.

中文翻译：

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血小板膜包被的金属-有机骨架纳米粒子在体内靶向基因沉默。

小干扰RNA（siRNA）是用于基因沉默的强大工具，已被广泛应用于生物医学领域，但其在体内的治疗用途面临许多挑战。在这里，我们报道了在体内靶向递送siRNA的血小板细胞膜包被的金属有机框架（MOF）纳米递送平台。MOF核心具有很高的负载量，其pH敏感性可在细胞摄取后破坏内体。细胞膜涂层提供了与疾病基质进行生物接口的天然方法。结果表明，可以在体外针对多个靶基因实现高沉默效率。使用鼠异种移植模型，观察到显着的抗肿瘤靶向性和治疗功效。总体，