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. Creative Biolabs is a world-class biotechnology company with an advanced gene therapy platform. Our objective is to provide optimal non-viral vectors for gene therapy to improve people's quality of life.

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. To eliminate the toxicity of polyplexes, Creative Biolabs has been devoted to developing decationized polyplexes that have no detrimental effects on the physicochemical stability or encapsulation ability. 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. Creative Biolabs provides commercial transfection reagents based on chitosan for therapeutic gene delivery.

Schematic diagram of chitosan as a gene delivery vector 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 cross-linked 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

Creative Biolabs has been devoted to improving the stability and efficiency of non-viral vectors by optimizing their structure, charge and formulation. We offer one-stop services about the development of polyplexes as a gene delivery system. Please feel free to contact us and let us know your needs.

Reference

  1. Novo, L.; et al. (2015). Decationized polyplexes for gene delivery.
For research use only. Not intended for any clinical use.