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Related Concept Videos

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...
Nucleic Acid Structure01:25

Nucleic Acid Structure

The pentose sugar in DNA is deoxyribose, while in RNA the pentose sugar is ribose. The difference between the sugars is the presence of the hydroxyl group on the ribose's second carbon and a hydrogen on the deoxyribose's second carbon. The phosphate residue attaches to the hydroxyl group of the 5′ carbon of one sugar and the hydroxyl group of the 3′ carbon of the sugar of the next nucleotide, which forms  a 5′ to 3′ phosphodiester linkage.
DNA Structure
DNA has a double-helix structure. The...
Site-Targeted Drug Delivery Systems: Polymeric Carriers01:24

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...
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...
Nucleic Acids02:43

Nucleic Acids

Nucleic acids are the most important macromolecules for the continuity of life. They carry the cell's genetic blueprint and carry instructions for its functioning.
DNA and RNA
The two main types of nucleic acids are deoxyribonucleic acid (DNA) and ribonucleic acid (RNA). DNA is the genetic material in all living organisms, ranging from single-celled bacteria to multicellular mammals. It is in the nucleus of eukaryotes and in the organelles, chloroplasts, and mitochondria. In prokaryotes, the...
Nucleic acids02:43

Nucleic acids

Nucleic acids are the most important macromolecules for the continuity of life. They carry the cell's genetic blueprint and carry instructions for its functioning.
DNA and RNA
The two main types of nucleic acids are deoxyribonucleic acid (DNA) and ribonucleic acid (RNA). DNA is the genetic material in all living organisms, ranging from single-celled bacteria to multicellular mammals. It is in the nucleus of eukaryotes and in the organelles, chloroplasts, and mitochondria. In prokaryotes, the...

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Related Experiment Video

Updated: May 14, 2026

Assembly and Characterization of Polyelectrolyte Complex Micelles
08:44

Assembly and Characterization of Polyelectrolyte Complex Micelles

Published on: March 2, 2020

Neutral polymeric micelles for RNA delivery.

Brittany B Lundy1, Anthony Convertine, Martina Miteva

  • 1Department of Bioengineering, University of Washington, Seattle, WA 98195, USA.

Bioconjugate Chemistry
|January 31, 2013
PubMed
Summary

This study presents a novel ampholytic polymer micelle system for effective RNA interference (RNAi) drug delivery. The developed system demonstrates high efficacy in gene silencing with minimal toxicity, paving the way for advanced RNAi therapeutics.

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Assembly and Characterization of Polyelectrolyte Complex Micelles
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Synthesis and Characterization of mRNA-Loaded Poly(Beta Aminoesters) Nanoparticles for Vaccination Purposes

Published on: August 13, 2021

Area of Science:

  • Biotechnology
  • Polymer Chemistry
  • Molecular Biology

Background:

  • RNA interference (RNAi) therapeutics hold great promise but require efficient and safe delivery systems.
  • Existing delivery methods often face challenges with efficacy and toxicity.
  • Development of advanced carriers is crucial for unlocking the full potential of RNAi drugs.

Purpose of the Study:

  • To develop and characterize a novel neutral, ampholytic polymeric delivery system for RNA interference (RNAi) drugs.
  • To evaluate the efficacy and biocompatibility of the developed system for siRNA delivery.
  • To demonstrate the potential of this carrier design for therapeutic applications.

Main Methods:

  • Synthesis of a diblock copolymer poly[(HPMA-co-PDSMA)-b-(PAA-co-DMAEMA-co-BMA)] using RAFT polymerization.
  • Self-assembly of diblock copolymers into polymeric micelles characterized by dynamic light scattering and fluorescence.
  • Conjugation of thiolated siRNA to polymeric micelles via thiol-disulfide exchange.
  • Evaluation of gene silencing efficacy (mRNA and protein knockdown) and toxicity in HeLa cells.

Main Results:

  • Polymeric micelles formed with a hydrodynamic radius of 25 nm and a critical micelle concentration of 25 μg/mL.
  • Efficient conjugation of siRNA to micelles, achieving 90% mRNA and 65% protein knockdown at a 1:10 siRNA to polymer ratio.
  • Demonstrated potent membrane destabilizing activity at endosomal pH, crucial for endosomal escape.
  • Negligible toxicity observed with the developed siRNA-polymer conjugates.
  • Comparison with micelles lacking a pH-responsive segment showed significantly reduced gene silencing.

Conclusions:

  • The developed neutral, ampholytic polymeric micelles represent a promising delivery system for therapeutic siRNA.
  • The system exhibits high efficacy in gene silencing and excellent biocompatibility.
  • The pH-responsive endosomalytic segment is critical for achieving potent therapeutic effects.
  • This carrier design offers a viable strategy for advancing RNAi drug delivery.