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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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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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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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Types of RNA01:23

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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.
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Nucleic Acids02:43

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Nucleic acids are the most important macromolecules for the continuity of life. They carry the cell's genetic blueprint and carry instructions for its functioning.
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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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Updated: Jun 23, 2025

Using In Vitro and In-cell SHAPE to Investigate Small Molecule Induced Pre-mRNA Structural Changes
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RNATACs: Multispecific small molecules targeting RNA by induced proximity.

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Cell Chemical Biology
|June 14, 2024
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Summary
This summary is machine-generated.

RNA-targeting chimeras (RNATACs) are novel small molecules that recruit RNA effectors to target RNA, offering new therapeutic potential. This approach expands druggable targets and could treat diseases with unmet needs.

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Area of Science:

  • Biochemistry
  • Molecular Biology
  • Drug Discovery

Background:

  • RNA-targeting small molecules (rSMs) offer novel therapeutic strategies by targeting RNA.
  • Traditionally undruggable proteins can be targeted by expanding the druggable space.
  • RNA-targeting chimeras (RNATACs) are an emerging class of molecules with therapeutic potential.

Purpose of the Study:

  • To review the potential advantages of RNATACs.
  • To summarize recent advancements in RNATAC technology.
  • To discuss the challenges and future directions of RNATACs.

Main Methods:

  • Review of current literature on RNA-targeting small molecules and RNATACs.
  • Analysis of the mechanism of action for RNATACs.
  • Discussion of potential applications and limitations.

Main Results:

  • RNATACs induce proximity between target RNA and RNA effectors (e.g., RNases).
  • RNATACs can modulate target RNA stability, localization, translation, or splicing.
  • This technology has broad potential for treating diseases with unmet needs.

Conclusions:

  • RNATACs represent a promising new modality in drug discovery.
  • Further research is needed to overcome challenges and realize the full potential of RNATACs.
  • This technology could expand therapeutic options for various diseases.