Innovative bile acid-cationic polymer nanoparticles in gene delivery: Cellular transfection relevant to eye, ear, and kidney cells

Gene therapy is an emerging treatment approach rapidly gaining broad interest across various pathologies. One common pitfall with gene therapy is the challenge of effective delivery, as current modalities suffer from poor safety and efficacious profiles. Polymer nanoparticles represent a novel method for the delivery of genes. Accordingly, this study developed novel polymer nanoparticles for the effective delivery of a reporter plasmid. Polymer nanoparticles were prepared by an ionotropic gelation technique with sodium alginate and cationic polymers; poly-L-lysine and chitosan. Bile acid excipients were incorporated for their therapeutic and permeation-enhancing properties. The nanoparticles were analyzed for physical properties including size, and charge as well as their safety and efficacy profiles. Eight nanoparticle formulations were prepared in the present study. Generated particles had a negative charge and particle size of between 100 and 400 nm. Particles also showed good biocompatibility against the cell lines tested, with acceptable survival at 0.5 mg/ml treatment concentration. Transfection efficiency however remained challenging. The addition of bile acid did improve transfection efficiency in some of the cell lines evaluated. Although suitable safety and physical characteristics were demonstrated in the investigated nanoparticles, further work involving formulation optimisation is required to improve transfection efficiency.