Protein is expressed in all major organs after intravenous infusion of mRNA-lipid nanoparticles in swine.

ABSTRACT: In vivo delivery of mRNA is promising for the study of gene expression and the treatment of diseases. Lipid nanoparticles (LNPs) enable efficient delivery of mRNA constructs, but protein expression has been assumed to be limited to the liver. With specialized LNPs, delivery to extrahepatic tissue occurs in small animal models; however, it is unclear if global delivery of mRNA to all major organs is possible in humans because delivery may be affected by differences in innate immune response and relative organ size. Furthermore, limited studies with LNPs have been performed in large animal models, such as swine, due to their sensitivity to complement activation-related pseudoallergy (CARPA). In this study, we found that exogenous protein expression occurred in all major organs when sw

SUBMITTER: Ferraresso F 

PROVIDER: S-EPMC11382111 | biostudies-literature | 2024 Sep

REPOSITORIES: biostudies-literature