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MicroRNAs01:22

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MicroRNA (miRNA) are short, regulatory RNA transcribed from introns—non-coding regions of a gene—or intergenic regions—stretches of DNA present between genes. Several processing steps are required to form biologically active, mature miRNA. The initial transcript, called primary miRNA (pri-mRNA), base-pairs with itself forming a stem-loop structure. Within the nucleus, an endonuclease enzyme, called Drosha, shortens the stem-loop structure into hairpin-shaped pre-miRNA. After...
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Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
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Macro and small over micro: macromolecules and small molecules that regulate microRNAs.

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Identifying microRNA (miRNA) regulators is crucial for treating diseases linked to miRNA changes. This review covers new oligonucleotide and small-molecule miRNA regulators and screening assays.

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Area of Science:

  • Biochemistry
  • Molecular Biology
  • Genetics

Background:

  • MicroRNAs (miRNAs) play critical roles in gene regulation.
  • Deregulation of specific miRNAs is associated with various disease states.
  • Targeted control of disease-associated miRNAs is a key therapeutic strategy.

Purpose of the Study:

  • To provide an updated overview of miRNA regulators.
  • To discuss oligonucleotide- and small-molecule-based therapeutic approaches for miRNA control.
  • To review assays for screening small-molecule miRNA regulators.

Main Methods:

  • Literature review of recent advancements in miRNA regulator research.
  • Analysis of different classes of miRNA regulators (oligonucleotides and small molecules).
  • Discussion of high-throughput screening (HTS) assays for small-molecule discovery.

Main Results:

  • Oligonucleotide-based regulators offer sequence-specific targeting of miRNAs.
  • Small-molecule regulators provide alternative therapeutic strategies with potential for oral administration.
  • Various screening assays have been developed to identify potent and selective small-molecule miRNA modulators.

Conclusions:

  • Effective miRNA regulators are essential for developing novel therapeutics against miRNA-mediated diseases.
  • Both oligonucleotide and small-molecule approaches show promise in modulating miRNA function.
  • Advancements in screening assays are crucial for the efficient discovery of small-molecule miRNA regulators.