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Generation of Cationic Nanoliposomes for the Efficient Delivery of In Vitro Transcribed Messenger RNA
Published on: February 1, 2019
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Human β-Defensin 23 as a Carrier for In Vitro and In Vivo Delivery of mRNA
Kyoung-Ran Kim1, Junghyun Kim1, Seunghye Cho2
1Chemical and Biological Integrative Research Center, Institute of Science and Technology (KIST), Hwarangno 14-gil 5, Seongbuk-gu, Seoul 02792, Republic of Korea.
Pharmaceutics
|October 28, 2023
Summary
A novel human peptide, hBD23, effectively delivers messenger RNA (mRNA) into cells. This biocompatible platform enhances mRNA therapeutics by improving cellular uptake and stability, showing promise for in vitro and in vivo applications.
Area of Science:
- Biotechnology
- Molecular Biology
- Drug Delivery Systems
Background:
- Effective intracellular delivery of messenger RNA (mRNA) is crucial for therapeutic applications but faces challenges due to mRNA's polyanionic nature, shape, and susceptibility to nucleases.
- Current delivery methods often struggle with efficiency, stability, and potential toxicity or immunogenicity.
- Developing biocompatible and efficient mRNA delivery vectors remains a significant hurdle in advancing mRNA therapeutics.
Purpose of the Study:
- To introduce and evaluate a novel mRNA delivery platform based on the human β-defensin peptide, hBD23.
- To assess the ability of hBD23/mRNA nanocomplexes to facilitate cellular uptake and protect mRNA from degradation.
- To determine the efficacy and biocompatibility of the hBD23 platform for both in vitro and in vivo mRNA delivery.
Main Methods:
- Formation of nanocomplexes between the positively charged hBD23 peptide and negatively charged mRNA.
- Optimization of peptide-to-mRNA (N/P) ratios to achieve efficient complex formation and delivery.
- In vitro and in vivo studies to evaluate cellular uptake, mRNA protection, protein expression, cytotoxicity, and immunogenicity.
Main Results:
- hBD23 successfully formed nanocomplexes with mRNA, enhancing cellular uptake and providing protection against serum nucleases.
- Optimized hBD23/mRNA complexes demonstrated efficient mRNA delivery and subsequent protein expression in vitro and in vivo.
- The human-derived nature of hBD23 resulted in minimal observed cytotoxicity and immunogenicity.
Conclusions:
- The human β-defensin peptide hBD23 serves as a highly effective and biocompatible platform for mRNA delivery.
- hBD23/mRNA nanocomplexes overcome key challenges in mRNA therapeutics, including cellular uptake and nuclease resistance.
- This novel delivery system holds significant promise for advancing the clinical application of mRNA-based therapies.
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