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mRNA-based therapies: Preclinical and clinical applications
Musga Qureischi1, Julia Mohr1, Estibaliz Arellano-Viera2
1BioNTech SE, Mainz, Germany.
Messenger RNA (mRNA) therapeutics deliver genetic instructions for protein production without altering DNA. This review explores mRNA
Area of Science:
- Biotechnology and Pharmaceutical Sciences
- Molecular and Cellular Biology
Background:
- Messenger RNA (mRNA) therapeutics offer a versatile platform for protein production.
- In vitro-transcribed (IVT) mRNA avoids nuclear entry, mitigating risks of genomic integration and insertional mutagenesis.
- Scalable manufacturing and customizable properties position mRNA for diverse therapeutic strategies.
Purpose of the Study:
- To review the advantages and applications of in vitro-transcribed (IVT) mRNA therapeutics.
- To discuss chemical modifications and delivery methods for optimizing mRNA therapies.
- To highlight preclinical and clinical applications of mRNA technology.
Main Methods:
- Review of existing literature on in vitro-transcribed (IVT) mRNA technology.
- Analysis of chemical modifications, purification techniques, and delivery systems for mRNA.
- Examination of preclinical and clinical data for various mRNA-based therapeutic applications.
Main Results:
- IVT mRNA can encode virtually any protein and is translated in the cytoplasm.
- mRNA production is scalable and less complex than recombinant protein production.
- mRNA-based vaccines demonstrated high efficacy during the COVID-19 pandemic.
Conclusions:
- IVT mRNA offers a safe, scalable, and adaptable platform for therapeutic protein production.
- Optimized mRNA sequence elements, modifications, and delivery enhance therapeutic potential.
- mRNA technology holds promise for vaccines, cancer immunotherapy, protein replacement, gene editing, and cell reprogramming.
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