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MicroRNA expression modulation

Molecular classification
Other (biological process; not a defined molecular family)
01

Overview

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.

Other names
miRNA expression regulationModulation of microRNA levelsRegulation of microRNA abundance
02

Mechanism of action

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)

03

Biological functions

Gene expression regulationPost-transcriptional regulationCell signalingEpigenetic control
04

Disease associations

Cancer (miRNA dysregulation is implicated in oncogenesis)Neurodegenerative diseaseCardiovascular diseaseInflammationInfectionOther (miRNA expression changes are associated with a wide spectrum of diseases)
05

Safety considerations

Off-target effects (modulation may impact multiple unintended mRNAs)Immune activation (delivery of oligonucleotide drugs or mimics can stimulate innate immune responses)Delivery challenges (cell- and tissue-specific delivery, nucleic acid stability)
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Interacting drugs

Antagomirs (chemically modified antisense oligonucleotides designed to inhibit specific miRNAs)

2 more in the full profile.

07

Biomarkers

Circulating miRNAs (tumor- or disease-associated miRNAs measured in blood, cerebrospinal fluid, etc.)Tissue miRNA expression profiles (used in cancer classification, prognosis, and response prediction)

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