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Development of Polyplexes as Gene Delivery System Gene therapy has emerged as an attractive modality for the treatment or prevention of acquired and genetic diseases. Non-viral gene delivery systems have great promise due to their ease of manufacturing and favorable immunogenicity as compared to viral vectors. Polyplexes based on polycationic structures have the ability to interact with negatively charged nucleic acids to spontaneously form nanoparticles. Thanks to their high loading capacity and ease of fabrication, several polyplexes have been extensively studied for the development of gene delivery therapeutics. Furthermore, the size, shape and surface chemistry of polyplexes vectors are modified to improve the targeting capability.

Poly (ethylene imine) (PEI) is one of the most potent polymers for gene delivery both “in vitro” and “in vivo”. Conjugation of poly (ethylene glycol) to PEI to form copolymers has been used to reduce the toxicity of PEI and provide steric stability to the complex. •

Chitosan Chitosan is a biodegradable polysaccharide copolymer with a positive charge. Chitosan as a vector has the properties of biocompatibility, safety and non-toxicity, protects the DNA from nuclease degradation and promotes the uptake.

Fig.1 Schematic diagram of chitosan as a gene delivery vector


Poly (lactic-co-glycolic acid) and poly lactic acid Poly (lactic-co-glycolic acid) (PLGA) and poly lactic acid (PLA) consist of lactic acid and glycolic acid. Biodegradable PLGA and PLA provide sustained gene delivery by bulk hydrolysis and increase stability.

Structural design of decationized polyplexes Decationized polyplexes are neutral polymers composed of a PEG corona and a disulfide crosslinked poly (hydroxypropyl methacrylamide) (pHPMA) core to encapsulate and retain the nucleic acids. Decationized polyplexes can function as stable and safe vectors to improve biodistribution for systemic gene therapy. Advantages:

No interference with cell viability

Excellent colloidal stability

Excellent cytocompatibility

Excellent safety profile

Low teratogenicity and mortality profile

Reference 1. Novo, L.; et al. (2015). Decationized polyplexes for gene delivery.

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Development of Polyplexes as Gene Delivery System  

Polyplexes based on polycationic structures have the ability to interact with negatively charged nucleic acids to spontaneously form nanopar...

Development of Polyplexes as Gene Delivery System  

Polyplexes based on polycationic structures have the ability to interact with negatively charged nucleic acids to spontaneously form nanopar...

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