Esposito Carla L, Catuogno Silvia, de Franciscis Vittorio
Istituto di Endocrinologia ed Oncologia Sperimentale, CNR, Via T. de Amicis 95, 80145, Naples, Italy.
Methods Mol Biol. 2016;1364:197-208. doi: 10.1007/978-1-4939-3112-5_16.
microRNAs (miRNAs) are short noncoding RNAs that effectively regulate the expression of a wide variety of genes. Increasing evidences have shown a fundamental role of miRNAs in cancer initiation and progression, thus indicating these molecules among the most promising for new approaches in cancer therapy. However, several hurdles limit the translation of miRNAs into the clinic. One of the most critical aspects is represented by the lack of a safe and reliable way to selectively target organs and tissues. Therefore, the development of cell-specific delivery means has become an essential step for the translation of miRNA-based therapeutics to clinic for cancer management. To this end aptamer-based approaches may provide efficient delivery tools for the selective accumulation of miRNA to target tumors, their intracellular uptake, processing, and functional silencing of target genes. In this chapter, we discuss the direct conjugation of miRNAs to aptamers against transmembrane receptors as innovative experimental approach for their selective delivery to cancer cells.
微小RNA（miRNA）是短链非编码RNA，可有效调控多种基因的表达。越来越多的证据表明，miRNA在癌症的发生和发展中起着重要作用，这表明这些分子是癌症治疗新方法中最有前景的分子之一。然而，若干障碍限制了miRNA在临床上的应用。最关键的方面之一是缺乏一种安全可靠的方法来选择性地靶向器官和组织。因此，开发细胞特异性递送方法已成为将基于miRNA的疗法应用于癌症治疗临床实践的关键步骤。为此，基于适配体的方法可为miRNA选择性积聚到靶肿瘤、其细胞内摄取、加工以及靶基因的功能沉默提供有效的递送工具。在本章中，我们将讨论miRNA与针对跨膜受体的适配体的直接缀合，这是一种将它们选择性递送至癌细胞的创新实验方法。
