Characterization of spray dried powders with nucleic acid-containing PEI nanoparticles.

摘要: Abstract Localized aerosol delivery of gene therapies is a promising treatment severe pulmonary diseases including lung cancer, cystic fibrosis, COPD and asthma. The administration drugs by inhalation features multiple benefits an enhanced patient acceptability compliance. application spray dried powder formulation has advantages over solutions due to their increased stability shelf life. Furthermore, optimal sizes the can be obtained drying allow deep deposition. present study optimized parameters involved with polyplexes formed polyethylenimine (PEI) nucleic acids in inert excipients generate nano-embedded microparticle (NEM) appropriate aerodynamic diameter. effects excipient matrix used NEM on biological activity acid ability recover embedded nanoparticles was investigated. showed that bioactivity integrity preserved after drying, could reconstituted from dry powders made trehalose but not mannitol as stabilizer. Scanning electron microscopy (SEM) formulations fused, lightly corrugated spherical particles range between 1 5 µm, while had smooth surfaces consisted more defined particles. After redispersion microparticles water, polyplex dispersions are comparable initial before drying. Cellular uptake transfection studies conducted adenocarcinoma cells show redispersed performed similar or better than were dried. A method for quantifying polymer loss following developed order ensure equal amounts all vitro experiments. results confirm containing prepared characteristics known therapy.