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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 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 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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Updated: Feb 17, 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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Cyclodextrin-siRNA conjugates as versatile gene silencing agents.

Meenakshi Malhotra1, Matt Gooding2, James C Evans3

  • 1Pharmacodelivery group, School of Pharmacy, University College Cork, Cork, Ireland; Department of Radiology, Stanford University School of Medicine, Stanford, CA 94305, USA.

European Journal of Pharmaceutical Sciences : Official Journal of the European Federation for Pharmaceutical Sciences
|December 2, 2017
PubMed
Summary

This study shows that functional small interfering RNAs (siRNAs) conjugated to β-cyclodextrin retain gene knockdown activity in cancer cells. Targeted nanoparticle delivery enhanced this gene silencing effect.

Keywords:
CyclodextrinsGene deliveryGlioblastomaNanoparticlesProstate cancerSiRNA conjugate

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Area of Science:

  • Biotechnology
  • Molecular Biology
  • Nanomedicine

Background:

  • Small interfering RNAs (siRNAs) are crucial for gene silencing.
  • β-cyclodextrin (β-CD) is a versatile host molecule for drug delivery.
  • Effective delivery of siRNA to cancer cells remains a challenge.

Purpose of the Study:

  • To assess the gene knockdown activity of siRNA conjugated to β-cyclodextrin.
  • To evaluate different formulations for delivering these siRNA-β-CD conjugates.
  • To demonstrate the viability of cyclodextrin-siRNA conjugates for gene silencing.

Main Methods:

  • Conjugation of functional siRNAs (luciferase and PLK1) to β-cyclodextrin.
  • Delivery of conjugates to cancer cell lines (U87, PC3, DU145) using polycation formulations (Lipofectamine 2000, cationic cyclodextrin).
  • Formation of ligand-targeted nanoparticles using adamantyl-PEG-ligands and chitosan for enhanced delivery.

Main Results:

  • siRNA-β-CD conjugation did not impair gene silencing efficacy.
  • Polycation formulations demonstrated successful gene knockdown.
  • Ligand-targeted nanoparticle formulations achieved enhanced gene silencing compared to non-targeted methods.

Conclusions:

  • Simple siRNA-β-CD conjugates are effective for gene silencing in cancer cells.
  • Targeted nanoparticle delivery systems significantly improve gene knockdown efficiency.
  • This approach demonstrates the potential of cyclodextrin-based siRNA conjugates for therapeutic applications.