期刊
METHODS AND PROTOCOLS
卷 4, 期 1, 页码 -出版社
MDPI
DOI: 10.3390/mps4010010
关键词
miRNA; miRNA delivery; miRNA therapeutics; viral vectors; non-viral vectors
miRNAs play crucial roles in regulating gene expression and their dysregulation is linked to various human pathologies. Utilizing carriers for efficient and safe delivery of miRNA-based therapeutics is essential for their clinical success.
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.
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