Specific mature microRNA (miRNA)

Mature microRNAs (miRNAs) are small, non-coding RNA molecules, typically 22 nucleotides in length, that regulate gene expression post-transcriptionally by binding to the 3' untranslated regions of target messenger RNAs (mRNAs). This binding typically results in gene silencing through translational inhibition or mRNA degradation (Bartel, 2004). miRNAs are involved in nearly every biological process, including development, cell proliferation, and apoptosis, and their dysregulation is a hallmark of various pathologies such as cancer, cardiovascular disease, and viral infections (Rupaimoole & Slack, 2017). As therapeutic targets, miRNAs offer the advantage of modulating entire gene networks rather than single proteins. Current pharmacological approaches include antagomirs (antisense oligonucleotides) to sequester overexpressed miRNAs and miRNA mimics to replenish downregulated ones. Notable examples in clinical development include Miravirsen for Hepatitis C and Cobomarsen for lymphoma (Janssen et al., 2013; Seto et al., 2018). However, the designation 'Specific mature microRNA' is a generic category rather than a specific molecular target, requiring further specification of the exact miRNA sequence for drug design.

Other names

Mature microRNAmiRNAmiRShort non-coding RNA

Inhibition of mature miRNA function via antisense oligonucleotides (antagomirs) or restoration of function via synthetic miRNA mimics (agomirs).