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Related Concept Videos

Site-Targeted Drug Delivery Systems: Polymeric Carriers01:24

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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...
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Assembly and Characterization of Polyelectrolyte Complex Micelles
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Recent developments in nucleic acid delivery with polyethylenimines.

Patrick Neuberg1, Antoine Kichler1

  • 1Laboratoire "Vecteurs: Synthèse et Applications Thérapeutiques", UMR7199 CNRS-Université de Strasbourg, Faculté de Pharmacie, Illkirch, France.

Advances in Genetics
|November 21, 2014
PubMed
Summary
This summary is machine-generated.

Polyethylenimines (PEIs) are effective nucleic acid delivery agents with a good safety profile in clinical trials. Their "proton sponge" mechanism enhances endosomal escape, making them versatile for gene therapy applications.

Keywords:
Antisense oligonucleotidesCationic polymersEndosomal releaseGene therapyNonviral gene deliveryPolyethyleniminesProton sponge effectRNA interference and siRNA

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Area of Science:

  • Biotechnology
  • Polymer Chemistry
  • Gene Therapy

Background:

  • Polyethylenimines (PEIs) are widely recognized for their efficiency in delivering nucleic acids.
  • Despite concerns regarding biodegradability, PEIs have demonstrated a favorable safety profile in clinical settings.

Purpose of the Study:

  • To elucidate the mechanism of PEI-mediated nucleic acid transfection.
  • To review recent advancements in the application of PEIs for delivering DNA, antisense oligonucleotides, and siRNA.

Main Methods:

  • Discussion of the "proton sponge" effect, a key mechanism for endosomal escape.
  • Overview of chemical modifications and conjugations possible with PEIs due to their amino groups.

Main Results:

  • PEIs exhibit high transfection efficiency attributed to their buffering capacity within endosomes.
  • The "proton sponge" effect facilitates endosomal escape of nucleic acid/PEI complexes (polyplexes).

Conclusions:

  • PEIs are versatile and efficient agents for nucleic acid delivery, with potential in gene therapy.
  • Ongoing research focuses on optimizing PEI-based delivery systems for therapeutic applications.