CORDIS Project
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This project focuses on improving gene therapy delivery systems using polyplexes, which are nanoscale carriers for therapeutic nucleic acids. By developing controlled assembly pathways for these carriers, the project aims to enhance the efficiency and effectiveness of gene delivery for treating genetic disorders.
A vast number of pathologies have a genetic origin.
Yet, using gene therapy to directly intervene at the genetic root remains a rarity within today’s pharmaceutical arsenal.
The main hurdles in promoting gene therapy from the lab to the clinic are the complex delivery pathways and biological instability of the therapeutic nucleic acids.
Polyplexes, nanoscale coacervates of nucleic acids and (synthetic) polymers, hold the prospect of being highly tunable, scalable and robust transport vehicles an…
UNIVERSITEIT UTRECHT
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