New mRNA technology turns cells into long-lasting drug factories

By delivering mRNA to cells that encode therapeutic proteins along with a signal peptide, the proteins could be transported to the endoplasmic reticulum and then secreted into the bloodstream. Credit: Lukas Farbiak; image created with

A team of researchers has established a ribonucleic acid (RNA)-based method that drives cells in the body to produce therapeutic proteins and secrete them into the bloodstream. The approach could potentially extend the lifespan of drugs in the body, reducing the burden on patients who require frequent drug administrations.

The researchers, based at UT Southwestern Medical Center, leveraged a naturally occurring biomolecule called a signal peptide that determines where cells send proteins—acting like a shipping label—to release proteins into the blood that would normally remain in the cell.

In their paper, published in Proceedings of the National Academy of Sciences, the new approach enabled secretion and increased the circulation time of a therapeutic protein compared to standard injections in a mouse model of psoriasis, demonstrating beneficial effects in these mice and separately, in animal models of cancer.

The modular nature of the technique suggests that it could be adapted to address a wide range of diseases.

“This could potentially be a powerful platform technology. By linking a signal peptide to any particular protein—insulin for patients with diabetes for example—you could very easily tailor this technique to different disease conditions,” said Jermont Chen, Ph.D., a program director in the National Institute of Biomedical Imaging and Bioengineering (NIBIB) Division of Discovery Science and Technology.




By National Institutes of Health

Article can be accessed on: