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A miRNA expression modulator refers to any molecule, compound, or biological agent that can alter the expression levels or activity of microRNAs (miRNAs) within a cell or organism. miRNAs are small (typically 18–25 nucleotides), non-coding RNA molecules that play a crucial role in post-transcriptional regulation of gene expression by binding to complementary sequences on target messenger RNAs (mRNAs), leading to mRNA degradation or translational repression. miRNA modulators influence the abundance, maturation, stability, or function of specific miRNAs, thereby indirectly regulating the expression of numerous genes targeted by those miRNAs. These modulators act by affecting either the transcription/processing of primary-miRNA transcripts, the biogenesis pathway (e.g., Drosha/Dicer processing), the loading into Argonaute-containing RISC complexes, or the stability/degradation rate of mature miRNAs. Targeting specific microRNAs with modulators is an emerging therapeutic strategy for diseases where dysregulated gene silencing plays a role. Modulators can be small molecules, antisense oligonucleotides (“antagomirs”), synthetic mimics (“agomirs”), peptides/proteins, or viral proteins, and can either inhibit (downregulate) or enhance (upregulate) endogenous levels/activity of particular miRNAs.
Modulation of miRNA transcription, processing, RISC loading, or stability; modulation of miRNA-mRNA binding affinity.
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