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RNA Interference01:23

RNA Interference

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...
Experimental RNAi02:15

Experimental RNAi

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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Related Experiment Video

Updated: May 23, 2026

Protocol for the Solid-phase Synthesis of Oligomers of RNA Containing a 2'-O-thiophenylmethyl Modification and Characterization via Circular Dichroism
11:37

Protocol for the Solid-phase Synthesis of Oligomers of RNA Containing a 2'-O-thiophenylmethyl Modification and Characterization via Circular Dichroism

Published on: July 28, 2017

Optimizing antisense oligonucleotides using phosphorodiamidate morpholino oligomers.

Linda J Popplewell1, Alberto Malerba, George Dickson

  • 1School of Biological Sciences, Royal Holloway, University of London, London, UK. linda.popplewell@rhul.ac.uk

Methods in Molecular Biology (Clifton, N.J.)
|March 29, 2012
PubMed
Summary

Antisense oligonucleotides (AOs) offer a promising gene therapy for Duchenne muscular dystrophy (DMD) by correcting the dystrophin gene reading frame. This approach, enabling exon skipping, has shown therapeutic potential in preclinical studies and patient cells.

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Related Experiment Videos

Last Updated: May 23, 2026

Protocol for the Solid-phase Synthesis of Oligomers of RNA Containing a 2'-O-thiophenylmethyl Modification and Characterization via Circular Dichroism
11:37

Protocol for the Solid-phase Synthesis of Oligomers of RNA Containing a 2'-O-thiophenylmethyl Modification and Characterization via Circular Dichroism

Published on: July 28, 2017

Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids
09:04

Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids

Published on: September 21, 2017

Area of Science:

  • Genetics
  • Molecular Biology
  • Biochemistry

Background:

  • Duchenne muscular dystrophy (DMD) results from mutations in the DMD gene that disrupt the protein's reading frame.
  • Restoring the reading frame can lead to a functional, albeit internally deleted, dystrophin protein, similar to Becker muscular dystrophy.

Purpose of the Study:

  • To explore the potential of antisense oligonucleotides (AOs) as a gene therapy for DMD.
  • To describe methodologies for optimizing AOs, specifically phosphorodiamidate morpholino oligomers, for targeted exon skipping in the DMD gene.

Main Methods:

  • Utilizing antisense oligonucleotides (AOs) to bind targeted mRNA sequences and modify pre-mRNA splicing.
  • Designing AOs to induce exon skipping, thereby correcting the reading frame of the mutated DMD transcript.
  • Focusing on phosphorodiamidate morpholino oligomers (PMOs) for specific exon targeting.

Main Results:

  • AO-induced exon skipping has demonstrated the potential to produce functional truncated dystrophin.
  • Therapeutic efficacy has been shown in vitro and in vivo in animal models, as well as in DMD patient cells and muscle explants.
  • This approach is poised to be the first gene therapy for DMD in clinical trials.

Conclusions:

  • Antisense oligonucleotide-mediated exon skipping represents a significant advancement in DMD gene therapy.
  • Personalized medicine approaches may be required due to the diverse range of DMD-causing mutations across the DMD gene.
  • Optimization and clinical validation of specific AOs are crucial for widespread application.