Grant: Collaborative Research and Development Grants

Polyelectrolyte complexes are nanoparticles that form via electrostatic attraction between a polycation and polyanion. A particular class of these systems is used to introduce genetic material into cells when the polyanion is a nucleic acid sequence like DNA or RNA. We have studied these delivery systems using the polycation chitosan, a natural polymer of glucosamine that is partly acetylated, and plasmid DNA to express genes when delivered to the nucleus. We have also developed nanoparticles containing siRNA which stop expression of the target gene. Patent-pending formulations of these nucleic acid delivery systems have commercial potential as transfection reagents to express proteins in cells or to silence genes through RNA interference and also as therapeutic agents that can be applied to animals and humans for different diseases. A critical aspect that needs to be addressed is scaled-up and quality controlled manufacturing to permit the generation of these products for sale as research reagents or as test articles in animal studies which are a prerequisite to human trials. The proposed project aims to complete development of fully documented manufacturing procedures so that the industry partners can proceed with further testing and marketing of these products to researchers in academia and industry who still require effective non-toxic reagents to deliver DNA and RNA to cells. A significant benefit to the industry partners and the scientific community will ensue by making these reagents available at a large scale. The results will be significant for the fields of nanotechnology, nucleic acid delivery, cell transfection, recombinant protein production, as well as gene silencing and gene therapy products.