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Experimental RNAi

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...

RNA Interference

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...

RNA Interference

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...

siRNA - Small Interfering RNAs

siRNA - Small Interfering RNAs

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.
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the ATP-dependent...

Site-Targeted Drug Delivery Systems: Polymeric Carriers

Site-Targeted Drug Delivery Systems: Polymeric Carriers

Polymeric carriers enhance targeted drug delivery by increasing efficacy while minimizing off-target effects. These carriers comprise a biodegradable polymeric backbone integrated with functional elements that enable targeting, improve physicochemical properties, and regulate drug release.Targeting MechanismsThe targeting ability of polymeric carriers is mediated by a homing device, which is a molecular recognition component designed to selectively bind to specific tissues or cells. Monoclonal...

Inhibitors of Viral Protein Synthesis

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Protein synthesis is indispensable for viral replication, as viruses lack the cellular machinery required for this process and must hijack the host's translational apparatus. In response, host cells deploy a critical innate immune defense involving interferons, specialized cytokines that play a central role in inhibiting viral propagation.Upon viral detection, infected cells release interferons that bind to receptors on adjacent uninfected cells, activating the JAK-STAT signaling pathway and...

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Related Experiment Video

Updated: Jun 24, 2026

Preparation of Neutrally-charged, pH-responsive Polymeric Nanoparticles for Cytosolic siRNA Delivery

Preparation of Neutrally-charged, pH-responsive Polymeric Nanoparticles for Cytosolic siRNA Delivery

Published on: May 2, 2019

Delivery of RNA interference therapeutics using polycation-based nanoparticles.

Kenneth Alan Howard¹

¹Interdisciplinary Nanoscience Center (iNANO), Department of Molecular Biology, University of Aarhus, 8000 Aarhus C, Denmark. kenh@inano.dk

Advanced Drug Delivery Reviews

|April 10, 2009

Summary

Polycation-based nanoparticles (polyplexes) effectively deliver RNA interference (RNAi) molecules for enhanced therapeutic efficacy. These versatile nanoparticles improve drug delivery and target interaction in animal models.

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Related Experiment Videos

Last Updated: Jun 24, 2026

Preparation of Neutrally-charged, pH-responsive Polymeric Nanoparticles for Cytosolic siRNA Delivery

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Published on: May 2, 2019

Delivery of Therapeutic siRNA to the CNS Using Cationic and Anionic Liposomes

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Published on: July 23, 2016

Polyethyleneimine-coated Iron Oxide Nanoparticles as a Vehicle for the Delivery of Small Interfering RNA to Macrophages In Vitro and In Vivo

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Published on: February 5, 2019

Area of Science:

Biotechnology
Nanomedicine
Molecular Therapeutics

Background:

RNA interference (RNAi) therapies require efficient delivery of RNA molecules.
Polycation-based nanoparticles (polyplexes) self-assemble with RNA to improve pharmacokinetics and intracellular trafficking.
Effective delivery is crucial for the therapeutic success of RNAi-based treatments.

Purpose of the Study:

To review the application of polyplexes for extracellular and intracellular delivery of synthetic RNA molecules.
To focus on routes of administration and gene silencing effects in animal disease models.
To discuss the incorporation of functional components for controlled cellular trafficking and RNA release.

Main Methods:

Review of literature on polycation-based nanoparticles for RNA delivery.
Analysis of studies detailing administration routes and efficacy in animal models.
Examination of strategies for incorporating functional components into nanoparticles.

Main Results:

Polyplexes demonstrate versatility in delivering RNA molecules for RNAi therapies.
Successful modulation of pharmacokinetics and intracellular trafficking by polyplexes.
Evidence of gene silencing effects in various animal disease models.

Conclusions:

Polycation-based nanoparticles offer a flexible platform for RNA delivery in RNAi therapeutics.
Nanoparticle design can be adapted to meet evolving delivery requirements for RNA drugs.
Polyplexes hold significant promise for advancing RNAi-based therapeutic strategies.