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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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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 RNA01:20

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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RNA Editing02:23

RNA Editing

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RNA editing is a post-transcriptional modification where a precursor mRNA (pre-mRNA) nucleotide sequence is changed by base insertion, deletion, or modification. The extent of RNA editing varies from a few hundred bases, in mitochondrial DNA of trypanosomes, to a just single base, in nuclear genes of mammals. Even a single base change in the pre-mRNA can convert a codon for one amino acid into the codon for another amino acid or a stop codon. This type of re-coding can significantly affect the...
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Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids
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Antisense RNA Therapeutics: A Brief Overview.

Virginia Arechavala-Gomeza1,2, Alejandro Garanto3,4,5

  • 1Ikerbasque, Basque Foundation for Science, Bilbao, Spain. virginia.arechavalagomeza@osakidetza.eus.

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Antisense oligonucleotides are versatile nucleic acid therapeutics targeting RNA. These molecules show promise for treating rare genetic diseases and common disorders, advancing from research tools to clinical applications.

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Area of Science:

  • Biotechnology
  • Molecular Biology
  • Genetics

Background:

  • Nucleic acid therapeutics are an expanding field, highlighted by mRNA vaccine success.
  • Antisense oligonucleotides (ASOs) are RNA-based molecules targeting pre-mRNA and mRNA.
  • ASOs have transitioned from research tools to promising clinical therapeutics.

Purpose of the Study:

  • To describe the mechanisms of action for RNA therapeutic molecules.
  • To provide examples of ASO applications in preclinical and clinical research.

Main Methods:

  • Review of antisense oligonucleotide mechanisms.
  • Compilation of preclinical and clinical case studies.

Main Results:

  • ASOs offer versatile targeting of RNA molecules.
  • Demonstrated potential for personalized medicine in rare genetic disorders.
  • Development pipeline includes treatments for common diseases.

Conclusions:

  • Antisense oligonucleotides represent a significant advancement in nucleic acid therapeutics.
  • ASOs are increasingly recognized for their therapeutic potential across various diseases.
  • The field is rapidly evolving with promising preclinical and clinical developments.