Research Institute for Medicines and Pharmaceutical Sciences (iMed.UL), Faculty of Pharmacy, University of Lisbon, Av. Prof. Gama Pinto, 1649-003 Lisbon, Portugal.
Drug Discov Today. 2013 Mar;18(5-6):282-9. doi: 10.1016/j.drudis.2012.10.002. Epub 2012 Oct 11.
MicroRNAs (miRNAs) are pivotal post-transcriptional gene expression regulators. These endogenous small non-coding RNAs aberrantly expressed in cancer have significant roles in tumorigenesis and progression. Currently, miRNAs are being pursued as diagnostic and prognostic biomarkers, and as therapeutic tools in cancer. miRNA modulation provides the unique ability to fine-tune multiple genes simultaneously, thereby regulating relevant signaling pathways involved in cell differentiation, proliferation and survival. This unique miRNA feature shifts the traditional one drug one target paradigm to a novel one drug multiple targets paradigm. We herein review in vivo strategies of miRNA modulator (mimic and/or inhibitor) delivery in cancer models, a subject that remains the key challenge to the establishment of this novel class of RNA therapeutics.
MicroRNAs (miRNAs) 是重要的转录后基因表达调控因子。这些在癌症中异常表达的内源性小分子非编码 RNA 在肿瘤发生和进展中具有重要作用。目前，miRNAs 被作为诊断和预后生物标志物，以及癌症的治疗工具进行研究。miRNA 的调节提供了同时微调多个基因的独特能力，从而调节涉及细胞分化、增殖和存活的相关信号通路。miRNA 的这种独特特征将传统的一种药物针对一个靶标的模式转变为一种药物针对多个靶标的新模式。本文综述了 miRNA 调节剂（模拟物和/或抑制剂）在癌症模型中的体内递送策略，这一主题仍然是建立这种新型 RNA 治疗药物的关键挑战。
