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Using Lipid Nanoparticles for the Delivery of Chemically Modified mRNA into Mammalian Cells
Published on: June 10, 2022
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Efficient hepatic delivery and protein expression enabled by optimized mRNA and ionizable lipid nanoparticle
Tongren Yang1, Chunhui Li1, Xiaoxia Wang2
1School of Life Science, Advanced Research Institute of Multidisciplinary Science, Institute of Engineering Medicine, Key Laboratory of Molecular Medicine and Biotherapy, Beijing Institute of Technology, Beijing, 100081, China.
Bioactive Materials
|July 22, 2020
Summary
This study introduces a new ionizable lipid nanoparticle (iLNP) for delivering messenger RNA (mRNA) therapies. The iLNP platform successfully enabled protein expression in cells, showing promise for future mRNA therapeutics.
Area of Science:
- Biotechnology
- Molecular Biology
- Drug Delivery Systems
Background:
- Messenger RNA (mRNA) represents a promising therapeutic modality with applications in vaccines, protein replacement, and cancer immunotherapy.
- Efficient in vivo delivery and mRNA molecule optimization remain significant challenges for widespread mRNA therapeutic application.
Purpose of the Study:
- To develop and evaluate a novel ionizable lipid nanoparticle (iLNP) system for enhanced in vitro and in vivo mRNA delivery.
- To assess the efficacy of codon-optimized mRNA formulations using the developed iLNP platform.
Main Methods:
- Codon-optimized and unoptimized mRNAs encoding luciferase and erythropoietin (EPO) were synthesized via in vitro transcription.
- mRNA molecules were formulated with a novel ionizable lipid (iBL0713) to create iLP171/mRNA nanoparticles.
- Protein expression levels and efficiency were analyzed in human hepatocellular carcinoma cells and hepatocytes post-administration.
Main Results:
- Successful in vitro and in vivo expression of luciferase and EPO proteins was achieved using the iLP171/mRNA formulations.
- Peak protein expression was observed at 6 hours post-administration.
- Codon-optimized mRNA demonstrated significantly higher EPO expression efficiency compared to unoptimized mRNA.
- No significant toxicity or immunogenicity was detected for the developed mRNA formulations.
Conclusions:
- The developed iLNP platform, utilizing iBL0713 lipid, provides an effective system for mRNA delivery and protein expression.
- Codon optimization of mRNA enhances therapeutic protein production efficiency.
- This study presents a viable and promising platform for the advancement of mRNA-based therapeutics.
