核酸适配体介导的短RNA治疗在癌细胞中的选择性递送。

Carla Lucia Esposito, Silvia Catuogno, Vittorio de Franciscis
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引用次数: 0

摘要

RNA干扰(RNAi)是最终导致基因表达抑制的重要生物学过程。RNAi的激活剂通常是小干扰rna (siRNA)和microRNAs (miRNA),具有相当大的治疗潜力。然而,将这些分子用于临床的一个主要障碍是缺乏安全可靠的方法将它们特异性地递送到靶细胞。在这方面,一类非常有前途的分子是核酸适体。它们是短的、结构化的单链rna或dna寡核苷酸,通过与靶分子的高特异性结合,提供高亲和力的配体和疾病相关蛋白的潜在拮抗剂。此外,由于高结合特异性,适体是一种强大的工具,可以选择性地将包括mi/ sirna、化疗药物、毒素和纳米颗粒在内的治疗物质输送到癌细胞或组织,从而潜在地提高给定治疗的疗效并降低毒性。在这篇综述中,我们将重点介绍适配体介导的mi/siRNA递送领域的最新进展,讨论它们在癌症治疗中的潜力和挑战。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

Aptamer-mediated selective delivery of short RNA therapeutics in cancer cells.

Aptamer-mediated selective delivery of short RNA therapeutics in cancer cells.

Aptamer-mediated selective delivery of short RNA therapeutics in cancer cells.

Aptamer-mediated selective delivery of short RNA therapeutics in cancer cells.

RNA interference (RNAi) is an important biological process that ultimately leads to suppression of gene expression. Activators of RNAi are typically small interfering RNAs (siRNA) and microRNAs (miRNA) that offer considerable therapeutic potnetial. However, a major obstacle to take these these molecules to the clinic is the absence of safe and reliable means for their specific delivery to target cells. In this regard, a highly promising class of molecules is represented by nucleic acid aptamers. These are short, structured, single-stranded RNAs or DNAs oligonucleotides that, by binding with high specificity to target molecules, provide high affinity ligands and potential antagonists of disease-associated proteins. Further, because of the high binding specificity, aptamers represent a powerful tool for the selective delivery of therapeutic cargos, including mi/siRNAs, chemotherapeutics, toxins and nanoparticles to cancer cells or tissues, thus potentially increasing the efficacy of a given therapy as well as reducing toxicity. In this review, we will focus on recent advances in the field of aptamer-mediated mi/siRNA delivery, discussing their potential and challenges in cancer therapy.

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