Abstract 19616: M3-RNA Encapsulation Technology Enables Rapid and Temporally Restricted Protein Expression

Introduction: Targeted induction of protein synthesis by exogenous delivery of coding synthetic mRNA would provide a unique new resource for intervention. Here, an M3-RNA (Microencapsulated Modified Messenger RNA) approach was evaluated. This technology will enable us to attain rapid protein expression as mRNA is translated within the cytoplasm. Hypothesis: Using M3-RNA technology we can achieve targeted delivery of mRNA with temporally restricted protein expression. Methods: In vitro and in vivo transfection transfection using modified messenger RNA (mmRNA) was assessed utilizing nanoparticle sized liposomes. Results: In vitro protein expression was analyzed upon transfection with mCherry mRNA in multiple cell lines (HEK293, HUVEC, Human Dermal and Cardiac Fibroblasts, HL-1 Cardiomyocyte cell line). Rapid expression of reporter genes within the cells was seen in as early as 2-4 hours and lasted for as long as 7 days and transfection efficiency was assessed using flow cytometry. We found almost identical ...