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Related Concept Videos

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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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.
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

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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

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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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Types of RNA01:20

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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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Subviral Agents01:29

Subviral Agents

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Subviral agents are infectious entities that resemble viruses but lack one or more viral components, such as a capsid or essential replication machinery. These agents include viroids, prions, and satellites, each possessing distinct structural and functional characteristics that influence their mode of infection and replication.Viroids are the simplest subviral agents, consisting of circular, single-stranded RNA molecules without a protein coat. They exclusively infect plants, relying entirely...
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Related Experiment Video

Updated: Oct 7, 2025

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
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Non-viral vectors for RNA delivery.

Yi Yan1, Xiao-Yu Liu1, An Lu1

  • 1Beijing Key Laboratory of Molecular Pharmaceutics and New Drug Delivery Systems, State Key Laboratory of Natural and Biomimetic Drugs, School of Pharmaceutical Sciences, Peking University, Beijing, China.

Journal of Controlled Release : Official Journal of the Controlled Release Society
|January 12, 2022
PubMed
Summary

RNA therapeutics offer a novel approach to disease treatment. Advanced non-viral delivery systems, including lipid nanoparticles, enhance RNA protection and targeted delivery for improved therapeutic outcomes.

Keywords:
Biological barrierControl releaseGene therapyNon-viral vectorRNA drugs

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Area of Science:

  • Biotechnology
  • Molecular Biology
  • Nanomedicine

Background:

  • RNA-based therapies utilize exogenous nucleic acids like mRNA, siRNA, miRNA, or ASO to modulate gene expression.
  • The success of mRNA vaccines against COVID-19 highlights the potential of RNA therapeutics.

Purpose of the Study:

  • To review biological barriers in in vivo RNA delivery.
  • To discuss advancements in non-viral RNA delivery systems for therapeutic applications.

Main Methods:

  • Review of current literature on RNA delivery systems.
  • Analysis of non-viral nanocarriers for RNA protection and targeting.

Main Results:

  • Non-viral delivery systems effectively protect RNA from degradation by ribonucleases.
  • Nanoparticles facilitate targeted tissue accumulation, cellular uptake, and controlled release of RNA therapeutics.

Conclusions:

  • Non-viral delivery systems are crucial for overcoming in vivo RNA delivery challenges.
  • These advanced systems hold significant promise for the future of RNA-based disease treatment.