Engineered mRNA turns your body into a drug-making biofactory

Messenger RNA (mRNA) contains the instructions that direct a cell to make a specific protein using its built-in machinery. Many will be aware of mRNA because of its association with the COVID-19 vaccine. But mRNA has potential uses far beyond that, including as a gene-based treatment for a range of diseases.

A recently published study details one such use. Researchers from the University of Texas (UT) Southwestern Medical Center have used engineered mRNA to prompt cells to secrete their own drugs to successfully treat psoriasis and cancer in mice.

“Instead of going to the hospital or outpatient clinic frequently for infusions, this technology may someday allow a patient to receive a treatment at a pharmacy or even at home once a month, which would be a significant boost to their quality of life,” said Daniel Siegwart, professor of Biomedical Engineering and Biochemistry at UT Southwestern and the study’s corresponding author.

Coinciding with recent advances in mRNA research has been progress in the field of delivering therapeutics using nanoparticles. However, most of the research has been directed towards having cells generate proteins that can be used directly within cells or else to indirectly trigger cellular pathways like those needed for gene editing. In the current study, the researchers took a different approach, focusing on getting those important proteins out of the cells so they can exert therapeutic effects elsewhere in the body.

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