Dasgupta Ishani, Chatterjee Anushila
Horae Gene Therapy Center, Department of Pediatrics, University of Massachusetts Medical School, Worcester, MA 01605, USA.
Department of Immunology and Microbiology, University of Colorado School of Medicine, Aurora, CO 80045, USA.
Methods Protoc. 2021 Jan 20;4(1):10. doi: 10.3390/mps4010010.
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.
微小RNA（miRNA）是一类短链非编码调节RNA分子，它们以组织和时间特异性的方式产生，以协调转录后基因表达。miRNA与靶mRNA杂交，诱导翻译抑制或mRNA降解。功能研究表明，miRNA几乎参与了每一个生理过程，因此，miRNA失调与多种人类疾病相关。因此，分别恢复miRNA表达或下调异常表达的miRNA的miRNA模拟物和抗miRNA，是有效调控miRNA水平备受追捧的治疗策略。在这方面，促进基于miRNA的治疗药物高效、安全递送的载体对于这些药物的临床成功至关重要。在这里，我们强调了当前最先进的病毒和非病毒miRNA递送系统的优缺点，并就如何利用这些工具改善基于miRNA的治疗效果提供了观点。
