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Related Concept Videos

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.
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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.
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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

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.
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Nonsense-mediated mRNA Decay02:27

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The Upf proteins that carry out nonsense-mediated decay (NMD) are found in all eukaryotic organisms, including humans. Each protein has an individual role, but they need to work in collaboration. Upf1 is an ATP-dependent RNA helicase that unwinds the RNA helix. Because Upf1 can unwind any RNA, Upf2 and Upf3 are required to help Upf1 discriminate between nonsense and normal mRNAs.
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MicroRNAs01:22

MicroRNAs

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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: Aug 25, 2025

Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids
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Natural antisense transcripts as drug targets.

Olga Khorkova1, Jack Stahl1, Aswathy Joji1,2

  • 1Center for Therapeutic Innovation and Department of Psychiatry and Behavioral Sciences, University of Miami, Miami, FL, United States.

Frontiers in Molecular Biosciences
|October 17, 2022
PubMed
Summary
This summary is machine-generated.

New nucleic acid drugs can target non-coding RNA networks, particularly natural antisense transcripts (NATs), offering novel therapeutic strategies for various diseases. This review covers recent advancements in NAT-mediated therapies and drug technologies.

Keywords:
anisense oligonucleotideslong nocoding RNAnatural antisense transcript (NAT)nucleic acid based therapeuticsposttranscriptional regulation

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Area of Science:

  • Molecular Biology
  • Genetics
  • Pharmacology

Background:

  • Non-coding RNAs, especially natural antisense transcripts (NATs), regulate crucial cellular mechanisms.
  • These regulatory networks are implicated in diverse disease processes.
  • NATs offer promising targets for therapeutic intervention.

Purpose of the Study:

  • To review clinical and pre-clinical developments in NAT-mediated therapeutics.
  • To survey advancements in nucleic acid-based drug technologies enabling these therapies.

Main Methods:

  • Literature review of recent clinical and pre-clinical studies.
  • Analysis of emerging nucleic acid-based drug technologies.

Main Results:

  • Non-coding RNA networks, modulated by nucleic acid drugs, present new therapeutic avenues.
  • NATs are key players in disease-relevant biological mechanisms.
  • Significant progress is being made in harnessing NATs for therapeutic applications.

Conclusions:

  • NAT-mediated mechanisms are increasingly exploited for novel drug development.
  • Advances in nucleic acid drug technology are crucial for realizing the therapeutic potential of NATs.