Current gene therapyJul 26, 2012

Using mRNA to produce proteins from outside sources

mRNA Technology Weekly Brief ↗PubMed ↗DOI ↗

Updated Apr 2, 2026

Abstract

Synthetic messenger RNA is emerging as a powerful tool for transient protein expression in specific cells.

RNA vectors may offer advantages over traditional DNA-based vectors, particularly in safety and ethical considerations.
Cis-acting structural elements can enhance RNA stability and translation efficiency.
Recent developments focus on optimizing messenger RNA to improve protein expression modulation.
Current studies explore RNA-based vectors for applications in immunotherapy, T cell therapy, stem cell therapy, and gene therapy.

Simplified

Several viral and non-viral vectors have been developed for exogenous protein expression in specific cells. Conventionally, this purpose is achieved through the use of recombinant DNA. But mainly due to the risks associated with permanent genetic alteration of cells, safety and ethical concerns have been raised for the use of DNA-based vectors in human clinical therapy. In the last years, synthetic messenger RNA has emerged as powerful tool to deliver genetic information. RNA vectors exhibit several advantages compared to DNA and are particularly interesting for applications that require transient gene expression. RNA stability and translation efficiency can be increased by cis-acting structural elements in the RNA such as the 5'-cap, the poly(A)-tail, untranslated regions and the sequence of the coding region. Here we review recent developments in the optimization of messenger RNA as vector for modulation of protein expression emphasizing on stability, transfection and immunogenicity. In addition, we summarize current pre-clinical and clinical studies using RNA-based vectors for immunotherapy, T cell, stem cell as well as gene therapy.

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Using mRNA to produce proteins from outside sources

Abstract

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