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Related Experiment Video

Updated: Apr 14, 2026

Porous Silicon Microparticles for Delivery of siRNA Therapeutics
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Published on: January 15, 2015

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Protease-triggered siRNA delivery vehicles.

David B Rozema1, Andrei V Blokhin1, Darren H Wakefield1

  • 1Arrowhead Research Corporation, Arrowhead Madison, 465 Science Drive, Madison, WI 53711, USA.

Journal of Controlled Release : Official Journal of the Controlled Release Society
|April 19, 2015
PubMed
Summary

Researchers developed a novel method for delivering therapeutics into cells using protease-sensitive polymers. This approach ensures safe and effective cytoplasmic access for small interfering RNA (siRNA) delivery without cell lysis.

Keywords:
SubcutaneousTargeted deliverysiRNA polymer conjugate

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Area of Science:

  • Biotechnology
  • Polymer Chemistry
  • RNA Therapeutics

Background:

  • * Delivering membrane-impermeable therapeutics safely to the cytoplasm is challenging.
  • * Conventional methods can cause toxicity through non-specific cell membrane lysis.
  • * Endogenous proteases offer a potential trigger for controlled drug release.

Purpose of the Study:

  • * To develop a controlled cytoplasmic delivery mechanism for therapeutics.
  • * To achieve functional delivery of small interfering RNA (siRNA) using a protease-triggered system.
  • * To create masked polymer-siRNA delivery vehicles for targeted cellular uptake.

Main Methods:

  • * Synthesized protease-sensitive polymer masking reagents based on polyethylene glycol (PEG).
  • * Incorporated N-acetylgalactosamine for targeting hepatocytes via asialoglycoprotein receptors.
  • * Formulated masked polymer-siRNA delivery vehicles with controlled size, charge, and stability.

Main Results:

  • * Proteolytic hydrolysis of masked polymers restored membrane-disrupting activity upon endocytosis.
  • * Functional delivery of siRNA into cells was achieved after subcutaneous administration.
  • * The delivery system demonstrated potential for extrahepatic targeting with ligand modification.

Conclusions:

  • * A novel, protease-triggered system enables safe and effective cytoplasmic delivery of siRNA.
  • * Reversible polymer inhibition and protease-sensitive substrates allow controlled therapeutic release.
  • * This platform holds promise for targeted RNA therapeutics and broader applications beyond the liver.