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Related Concept Videos

RNA Interference01:23

RNA Interference

28.4K
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 RNA01:20

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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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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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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Viruses with RNA Genomes01:29

Viruses with RNA Genomes

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RNA viruses are categorized into positive-strand, negative-strand, or double-stranded groups based on their genomic structure and replication mechanisms. This classification dictates how they exploit host cellular machinery for protein synthesis and replication. Some RNA viruses also utilize reverse transcription as part of their life cycle, further diversifying their replication strategies.Positive-Strand RNA VirusesPositive-strand RNA viruses have genomes that function directly as messenger...
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Related Experiment Video

Updated: Mar 7, 2026

High-throughput Screening for Broad-spectrum Chemical Inhibitors of RNA Viruses
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Overcoming cellular barriers for RNA therapeutics.

Steven F Dowdy1

  • 1Department of Cellular and Molecular Medicine, University of California, San Diego, School of Medicine, La Jolla, California, USA.

Nature Biotechnology
|March 1, 2017
PubMed
Summary

RNA therapeutics show promise for treating diseases by targeting genes, but cell entry remains a challenge. Recent chemical advancements are beginning to overcome this barrier for effective RNA delivery.

Area of Science:

  • Biotechnology
  • Molecular Biology
  • Therapeutics

Background:

  • RNA-based therapies, including siRNA, miRNA, ASOs, aptamers, mRNA, and CRISPR-Cas9, offer novel treatment strategies for various diseases.
  • These RNA modalities hold potential for targeting previously undruggable genes and creating new therapeutic approaches.
  • A significant hurdle for RNA therapeutics is overcoming cellular defenses that prevent RNA entry into cells.

Purpose of the Study:

  • To highlight the potential of RNA-based therapeutics.
  • To identify the major challenge in RNA therapeutic development.
  • To discuss recent advancements in overcoming delivery barriers.

Main Methods:

  • Review of current RNA-based therapeutic modalities.
  • Analysis of biological barriers to intracellular RNA delivery.

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  • Examination of recent chemical advancements in RNA delivery systems.
  • Main Results:

    • RNA therapeutics offer a paradigm shift in treating diseases like cancer, Alzheimer's, and influenza.
    • The primary obstacle for RNA therapeutics is the inability to efficiently cross the cell membrane (lipid bilayer).
    • Recent chemical innovations are showing success in penetrating this barrier, improving RNA delivery.

    Conclusions:

    • RNA therapeutics have vast potential but require effective intracellular delivery.
    • Overcoming the cell membrane barrier is crucial for the widespread success of RNA-based medicines.
    • Emerging chemical strategies represent a significant step forward in RNA delivery technology.