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Related Concept Videos

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.
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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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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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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.
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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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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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Related Experiment Video

Updated: Jun 10, 2025

Protocol for the Solid-phase Synthesis of Oligomers of RNA Containing a 2'-O-thiophenylmethyl Modification and Characterization via Circular Dichroism
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Enhancing RNA inhibitory activity using clamp-G-modified nucleobases.

Sai Pallavi Pradeep1, Vikas Kumar1, Shipra Malik1

  • 1Department of Pharmaceutical Sciences, University of Connecticut, Storrs, CT 06269, USA.

Cell Reports. Physical Science
|October 18, 2024
PubMed
Summary

Clamp-G modified peptide nucleic acids (cGPNAs) show promise as microRNA and messenger RNA inhibitors. These cGPNAs effectively reduced tumor growth in lymphoma models and targeted transthyretin mRNA in vivo.

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Area of Science:

  • Oligonucleotide Therapeutics
  • Antisense Technology
  • Molecular Biology

Background:

  • MicroRNAs (miRNAs) and messenger RNAs (mRNAs) are key regulators of gene expression.
  • Dysregulation of miRNAs, such as miR-155, is implicated in diseases like diffuse large B cell lymphoma.
  • Peptide nucleic acids (PNAs) are DNA mimics with potential as therapeutic agents.

Purpose of the Study:

  • To evaluate clamp-G nucleobase-modified peptide nucleic acids (cGPNAs) as inhibitors of specific miRNAs and mRNAs.
  • To demonstrate the therapeutic potential of cGPNAs in preclinical models of lymphoma and liver disease.

Main Methods:

  • cGPNAs were designed to target miR-155 and transthyretin (TTR) mRNA.
  • In vitro studies assessed miRNA/mRNA downregulation and effects on downstream targets in lymphoma cell lines.
  • In vivo studies utilized xenograft mouse models for lymphoma and liver-targeted delivery of cGPNAs.

Main Results:

  • cGPNAs significantly downregulated miR-155 and upregulated its targets in lymphoma cells.
  • In vivo cGPNA treatment reduced tumor growth and improved survival in a lymphoma xenograft model.
  • Antisense cGPNAs demonstrated dose-dependent knockdown of TTR mRNA and protein in vivo, with enhanced liver delivery.

Conclusions:

  • Clamp-G modified PNA analogs represent a robust platform for antisense-based therapies.
  • cGPNAs show significant potential for treating miR-155-associated cancers and TTR-related liver conditions.
  • This study validates cGPNAs as a versatile and effective antisense modality.