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MicroRNA expression modulation refers to the alteration in the levels of microRNAs, a class of small, non-coding RNAs (~21 nucleotides) that regulate gene expression mainly through translational repression or degradation of target mRNAs. Changes in miRNA expression influence diverse biological processes such as development, cell proliferation, apoptosis, and differentiation, as well as pathological states including cancer and cardiovascular and neurodegenerative diseases. Modulation can occur physiologically in response to cellular signals or pathologically in disease states, and can be therapeutically targeted using antisense oligonucleotides, miRNA mimics, or small molecule drugs. Because miRNAs affect large networks of gene expression, the therapeutic modulation of their expression requires careful consideration of specificity, off-target effects, and safe delivery.
Inhibition of specific miRNAs (antagomirs bind to and degrade/inactivate target miRNAs) Replacement therapy (miRNA mimics restore function of downregulated miRNAs) Modulation of biogenesis (compounds can act on components of the miRNA processing machinery)
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