A Polymeric Nanoparticle Formulation for Targeted mRNA Delivery to Fibroblasts.

ABSTRACT: Messenger RNA (mRNA)-based therapies offer enhanced control over the production of therapeutic proteins for many diseases. Their clinical implementation warrants formulations capable of delivering them safely and effectively to target sites. Owing to their chemical versatility, polymeric nanoparticles can be designed by combinatorial synthesis of different ionizable, cationic, and aromatic moieties to modulate cell targeting, using inexpensive formulation steps. Herein, 152 formulations are evaluated by high-throughput screening using a reporter fibroblast model sensitive to functional delivery of mRNA encoding Cre recombinase. Using in vitro and in vivo models, a polymeric nanoformulation based on the combination of 3 specific monomers is identified to transfect fibroblasts much more effe

SUBMITTER: Rodrigues AF 

PROVIDER: S-EPMC9929262 | biostudies-literature | 2023 Feb

REPOSITORIES: biostudies-literature