Department of Life Sciences and Biotechnology, University of Ferrara, 44121 Ferrara, Italy.
Laboratory of Molecular Pathology, University-Hospital, 37126 Verona, Italy.
Molecules. 2017 Dec 29;23(1):71. doi: 10.3390/molecules23010071.
Peptide nucleic acids (PNAs) are very useful tools for gene regulation at different levels, but in particular in the last years their use for targeting microRNA (anti-miR PNAs) has provided impressive advancements. In this respect, microRNAs related to the repression of cystic fibrosis transmembrane conductance regulator (CFTR) gene, which is defective in cystic fibrosis, are of great importance in the development of new type of treatments. In this paper we propose the use of an anti-miR PNA for targeting miR-145, a microRNA reported to suppress CFTR expression. Octaarginine-anti-miR PNA conjugates were delivered to Calu-3 cells, exerting sequence dependent targeting of miR-145-5p. This allowed to enhance expression of the miR-145 regulated CFTR gene, analyzed at mRNA (RT-qPCR, Reverse Transcription quantitative Polymerase Chain Reaction) and CFTR protein (Western blotting) level.
肽核酸（PNA）是一种非常有用的工具，可用于在不同水平上调节基因，特别是在过去几年中，它们在针对 microRNA（抗-miR PNA）方面的应用取得了令人瞩目的进展。在这方面，与囊性纤维化跨膜电导调节因子（CFTR）基因抑制相关的 microRNAs 对于开发新型治疗方法非常重要，CFTR 基因在囊性纤维化中存在缺陷。在本文中，我们提出使用抗-miR PNA 来靶向 miR-145，这是一种被报道能抑制 CFTR 表达的 microRNA。八聚精氨酸-抗-miR PNA 缀合物被递送至 Calu-3 细胞，对 miR-145-5p 进行序列依赖性靶向。这使得 miR-145 调节的 CFTR 基因的表达得到增强，在 mRNA（RT-qPCR，逆转录定量聚合酶链反应）和 CFTR 蛋白（Western blot）水平进行了分析。
