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Related Concept Videos

RNA Interference01:23

RNA Interference

27.6K
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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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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Types of RNA01:20

Types of RNA

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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 regulating 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 Performs Diverse...
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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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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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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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How We Think about Targeting RNA with Small Molecules.

Matthew G Costales1, Jessica L Childs-Disney1, Hafeez S Haniff1

  • 1Department of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, Florida 33458, United States.

Journal of Medicinal Chemistry
|March 28, 2020
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Small molecules can effectively target RNA structures for drug development. New technologies enable precise targeting and degradation of functional RNAs, opening vast therapeutic possibilities.

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Area of Science:

  • Biochemistry
  • Medicinal Chemistry
  • Molecular Biology

Background:

  • RNA molecules possess structural diversity, offering significant potential as therapeutic targets for small molecule drugs.
  • Small molecules can selectively bind to and modulate the function of specific RNA structures.

Purpose of the Study:

  • To review the principles of molecular recognition between small molecules and RNA.
  • To highlight key technologies and properties enabling bioactive RNA-small molecule interactions.
  • To discuss the potential for small molecule-based RNA targeting in medicine.

Main Methods:

  • Exploration of sequence-based ligand design for RNA targets.
  • Analysis of methods for identifying small molecule binding sites on RNA.
  • Review of emerging technologies like ribonuclease-targeted chimeras (RIBOTACs) for RNA degradation.

Main Results:

  • Established rules for designing small molecule ligands that interact with RNA targets.
  • Methods to predict off-target interactions and identify functional RNA binding sites.
  • Development of novel strategies for small molecule-induced RNA degradation and cleavage.

Conclusions:

  • Small molecule-based targeting of RNA represents a promising frontier in drug discovery.
  • Advancements in molecular recognition and enabling technologies facilitate the development of RNA-targeted therapeutics.
  • The human transcriptome is a rich source of potential targets for small molecule chemical probes and medicines.