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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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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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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: Apr 20, 2026

Long-term Silencing of Intersectin-1s in Mouse Lungs by Repeated Delivery of a Specific siRNA via Cationic Liposomes. Evaluation of Knockdown Effects by Electron Microscopy
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Lipid nanoparticles for short interfering RNA delivery.

Alex K K Leung1, Yuen Yi C Tam1, Pieter R Cullis1

  • 1Department of Biochemistry and Molecular Biology, The University of British Columbia, Vancouver, BC, Canada.

Advances in Genetics
|November 21, 2014
PubMed
Summary

RNA interference (RNAi) therapeutics offer gene silencing for diseases. Lipid nanoparticle (LNP) delivery systems are key for effectively delivering short-interfering RNA (siRNA) to target cells, advancing clinical trials.

Keywords:
CRISPRGene silencingLipid nanoparticleMicrofluidicsRNA interferenceSystemic deliverymRNAsiRNA

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Area of Science:

  • Biotechnology
  • Molecular Biology
  • Pharmacology

Background:

  • RNA interference (RNAi) enables gene silencing for therapeutic applications.
  • Effective delivery of RNAi inducers like short-interfering RNA (siRNA) to target tissues is crucial for therapeutic success.
  • Lipid nanoparticle (LNP) technology has emerged as a leading strategy for systemic siRNA delivery.

Purpose of the Study:

  • To review challenges and delivery strategies for siRNA therapeutics.
  • To highlight the advancements in lipid nanoparticle (LNP) delivery systems for siRNA.
  • To explore the potential of LNP technology for delivering other nucleic acids.

Main Methods:

  • Review of existing literature on RNAi delivery systems.
  • Focus on lipid nanoparticle (LNP) formulation and development for siRNA.
  • Discussion of methods to enhance LNP-siRNA gene silencing efficacy.

Main Results:

  • LNP delivery systems are the most advanced for systemic siRNA delivery.
  • Numerous LNP-siRNA formulations are in various stages of clinical trials.
  • LNP technology shows promise for encapsulating mRNA and CRISPR for therapeutic applications.

Conclusions:

  • LNP technology is pivotal for advancing RNAi-based therapeutics.
  • Further development of LNP-siRNA enhances gene silencing potency.
  • LNP platforms have broad applications beyond siRNA delivery for nucleic acid therapeutics.