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Recent Advances in miRNA Delivery Systems

机译:MiRNA交付系统的最新进展

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摘要

MicroRNAs (miRNAs) represent a family of short non-coding regulatory RNA molecules that are produced in a tissue and time-specific manner to orchestrate gene expression post-transcription. MiRNAs hybridize to target mRNA(s) to induce translation repression or mRNA degradation. Functional studies have demonstrated that miRNAs are engaged in virtually every physiological process and, consequently, miRNA dysregulations have been linked to multiple human pathologies. Thus, miRNA mimics and anti-miRNAs that restore miRNA expression or downregulate aberrantly expressed miRNAs, respectively, are highly sought-after therapeutic strategies for effective manipulation of miRNA levels. In this regard, carrier vehicles that facilitate proficient and safe delivery of miRNA-based therapeutics are fundamental to the clinical success of these pharmaceuticals. Here, we highlight the strengths and weaknesses of current state-of-the-art viral and non-viral miRNA delivery systems and provide perspective on how these tools can be exploited to improve the outcomes of miRNA-based therapeutics.
机译:MicroRNA(miRNA)代表一种短的非编码调节RNA分子的系列,其以组织和时间特异性方式生产,以协调转录后的基因表达。 MiRNA杂交至靶mRNA以诱导翻译抑制或mRNA降解。功能性研究表明,MiRNA几乎每种生理过程从事,因此,miRNA脱少抑制与多种人类病理有关。因此,恢复miRNA表达或下调异常表达miRNA的miRNA模拟和抗miRNA是高度追捧的治疗策略,用于有效地操纵miRNA水平。在这方面,促进熟练和安全递送MiRNA治疗的载体载体是这些药物的临床成功的基础。在这里,我们突出了目前最先进的病毒和非病毒MiRNA递送系统的优点和缺点,并提供了如何利用这些工具来改善基于miRNA的治疗剂的结果的视角。

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