Aerosolizable Lipid Nanoparticles for Pulmonary Delivery of mRNA through Design of Experiments.

ABSTRACT: Messenger RNA is a class of promising nucleic acid therapeutics to treat a variety of diseases, including genetic diseases. The development of a stable and efficacious mRNA pulmonary delivery system would enable high therapeutic concentrations locally in the lungs to improve efficacy and limit potential toxicities. In this study, we employed a Design of Experiments (DOE) strategy to screen a library of lipid nanoparticle compositions to identify formulations possessing high potency both before and after aerosolization. Lipid nanoparticles (LNPs) showed stable physicochemical properties for at least 14 days of storage at 4 °C, and most formulations exhibited high encapsulation efficiencies greater than 80%. Generally, upon nebulization, LNP formulations showed increased particle size and de

SUBMITTER: Zhang H 

PROVIDER: S-EPMC7692784 | biostudies-literature | 2020 Oct

REPOSITORIES: biostudies-literature