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MicroRNAs01:22

MicroRNAs

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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 the pre-miRNA...
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siRNA - Small Interfering RNAs02:30

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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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RNA Interference01:23

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RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
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Experimental RNAi02:15

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RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...
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Genome-wide Screen for miRNA Targets Using the MISSION Target ID Library
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Genome-wide Screen for miRNA Targets Using the MISSION Target ID Library

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Targeting MicroRNAs with Small Molecules.

Kisanet Tadesse1, Raphael I Benhamou1

  • 1The Institute for Drug Research of the School of Pharmacy, Faculty of Medicine, The Hebrew University of Jerusalem, Jerusalem 91120, Israel.

Non-Coding RNA
|March 25, 2024
PubMed
Summary
This summary is machine-generated.

Small molecules are emerging as powerful tools to precisely target microRNAs (miRs) involved in diseases. This approach offers new therapeutic strategies for various conditions by modulating miR activity.

Keywords:
RIBOTACmicroRNAsmall moleculestargeted degradationtherapeutics

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Area of Science:

  • Biochemistry
  • Molecular Biology
  • Pharmacology

Background:

  • MicroRNAs (miRs) play crucial roles in cellular processes and are implicated in numerous diseases.
  • Targeting miRs presents an attractive strategy for developing novel therapeutics.
  • Developing precise methods to modulate miR activity is essential for effective treatment.

Purpose of the Study:

  • To review the progress in drug discovery for small molecules targeting microRNAs.
  • To highlight the potential of small molecules in modulating disease-associated miR function.
  • To discuss the transformative impact of these approaches on miR-targeted therapeutics and precision medicine.

Main Methods:

  • Systematic identification of small molecules capable of selectively targeting specific disease-associated miRs.
  • Leveraging multidisciplinary approaches in RNA research and drug discovery.
  • Developing compounds that can inhibit or degrade miRs to modulate their function.

Main Results:

  • Advances in RNA research enable the identification of specific small molecules for miR targeting.
  • Small molecules can effectively modulate the activity of disease-associated miRs.
  • These strategies are transforming the landscape of miR-targeted therapeutics.

Conclusions:

  • Small molecule-based modulation of miRs offers a potent therapeutic intervention.
  • This approach signifies a significant stride towards precision medicine for diverse diseases.
  • The multidisciplinary strategies discussed have the potential to revolutionize disease treatment.