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Related Concept Videos

RNA Interference01:23

RNA Interference

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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.
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...
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Experimental RNAi02:15

Experimental RNAi

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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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siRNA - Small Interfering RNAs02:30

siRNA - Small Interfering RNAs

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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.
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the...
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Types of RNA01:23

Types of RNA

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Overview
Three main types of RNA are involved in protein synthesis: messenger RNA (mRNA), transfer RNA (tRNA), and ribosomal RNA (rRNA). These RNAs perform diverse functions and can be broadly classified as protein-coding or non-coding RNA. Non-coding RNAs play important roles in the regulation of gene expression in response to developmental and environmental changes. Non-coding RNAs in prokaryotes can be manipulated to develop more effective antibacterial drugs for human or animal use.
RNA...
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Small interfering RNAs (siRNA)02:30

Small interfering RNAs (siRNA)

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Translational Regulation01:29

Translational Regulation

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Translational regulation in prokaryotes ensures efficient protein synthesis by controlling ribosome access to mRNA. This regulation is mediated by secondary RNA structures, including translational riboswitches, RNA thermometers, and small RNAs (sRNAs), which respond to intracellular and environmental signals to modulate gene expression.Translational RiboswitchesRiboswitches in the leader region of mRNAs can regulate translation by altering the accessibility of the Shine-Dalgarno (SD) sequence,...
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Related Experiment Video

Updated: Aug 14, 2025

Using In Vitro and In-cell SHAPE to Investigate Small Molecule Induced Pre-mRNA Structural Changes
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Using In Vitro and In-cell SHAPE to Investigate Small Molecule Induced Pre-mRNA Structural Changes

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Brief considerations on targeting RNA with small molecules.

Quentin Vicens1, Eric Westhof2

  • 1Department of Biochemistry and Molecular Genetics, RNA Bioscience Initiative, University of Colorado Anschutz Medical Campus, School of Medicine, Aurora, CO 80045, USA.

Faculty Reviews
|January 16, 2023
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Summary

RNA targeting offers therapeutic potential, but identifying targets and methods remains difficult. Recent advances in screening, optimization, and validation have enabled successful RNA-targeting drugs, with key factors guiding future breakthroughs.

Keywords:
Drug discoveryRNA structure & dynamicsRNA targetingSARS-CoV2

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Area of Science:

  • Biochemistry
  • Molecular Biology
  • Pharmacology

Background:

  • Ribonucleic acid (RNA) has been a recognized therapeutic target for over 30 years.
  • Challenges persist in identifying specific RNA targets and developing effective targeting strategies.
  • Recent progress has led to the approval of several RNA-targeting drugs.

Purpose of the Study:

  • To highlight key factors contributing to the success of RNA-targeting therapeutics.
  • To identify crucial aspects for future advancements in RNA-targeting drug development.

Main Methods:

  • Review of recent advances in screening, drug optimization, and target validation for RNA therapeutics.
  • Analysis of factors contributing to the clinical success of approved RNA-targeting drugs.

Main Results:

  • Confluence of screening, optimization, and validation approaches has yielded successful RNA-targeting therapeutics.
  • Several RNA-targeting drugs have received clinical approval.

Conclusions:

  • Key factors driving success in RNA-targeting drug development are identified.
  • Critical considerations for achieving future breakthroughs in RNA-targeting therapeutics are outlined.