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基于外泌体的纳米载体作为 RNA 干扰治疗载体的最新进展。

Recent advances in exosome-based nanovehicles as RNA interference therapeutic carriers.

机构信息

National Institute of Pharmaceutical Education & Research (NIPER) - Ahmedabad, Palaj, Opposite Air Force Station, Gandhinagar 382355, Gujarat, India.

TIT College of Pharmacy, Technocrats Institute of Technology Campus, Anand Nagar, Raisen Road, Bhopal 462021, Madhya Pradesh, India.

出版信息

Nanomedicine (Lond). 2017 Nov;12(21):2653-2675. doi: 10.2217/nnm-2017-0210. Epub 2017 Sep 29.

Abstract

RNA interference (RNAi) therapeutics (siRNA, miRNA, etc.) represent an emerging medicinal remedy for a variety of ailments. However, their low serum stability and low cellular uptake significantly restrict their clinical applications. Exosomes are biologically derived nanodimensional vesicle ranging from a few nanometers to a hundred. In the last few years, several reports have been published demonstrating the emerging applications of these exogenous membrane vesicles, particularly in carrying different RNAi therapeutics to adjacent or distant targeted cells. In this report, we explored the numerous aspects of exosomes from structure to clinical implications with special emphasis on their application in delivering RNAi-based therapeutics. siRNA and miRNA have attracted great interest in recent years due to their specific application in treating many complex diseases including cancer. We highlight strategies to obviate the challenges of their low bioavailability for gene therapy.

摘要

RNA 干扰 (RNAi) 疗法(siRNA、miRNA 等)是治疗多种疾病的新兴医学方法。然而,它们在血清中的稳定性低和细胞摄取率低,严重限制了它们的临床应用。外泌体是具有生物起源的纳米级囊泡,尺寸从几纳米到一百纳米不等。在过去几年中,已经有几篇报道发表,展示了这些外源性膜囊泡的新兴应用,特别是在将不同的 RNAi 疗法递送到相邻或远处的靶细胞方面。在本报告中,我们探讨了外泌体从结构到临床意义的众多方面,特别强调了它们在递送电镜 RNAi 疗法方面的应用。近年来,siRNA 和 miRNA 因其在治疗许多复杂疾病(包括癌症)方面的特殊应用而引起了极大的兴趣。我们强调了克服其基因治疗中生物利用度低的挑战的策略。

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