Scientists say they can now turn genes on and off like a light switch

Gene therapy is something most people would have heard of before, but what does it actually entail? Simply put, gene therapy is an innovative approach to medicine that uses genetic material to cure diseases.

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This genetic material is administered to the patient via the individual cells and, depending on the gene therapy, elicits the production of specific proteins. Diseases and medical afflictions can be cured, but the patient must stay within the therapeutic window. There are still some problems that need to be ironed out with gene therapy, such as being able to reliably stay within the therapeutic window. Too much of the protein may be toxic to the patient, and too little results in no therapeutic relief.

However, researchers from the Baylor College of Medicine penned a new study published in the journal Nature Biotechnology, which claims they have been able to regulate protein production, metaphorically similar to the dimming of a light switch in a bedroom. The team modified RNA molecules that are meant to produce the therapeutic protein and inserted a "stop sign" at the beginning of the gene's life. Additionally, the researchers bound a portion of the RNA with the FDA-approved antibiotic drug tetracycline.

So, here's how it works. When that particular portion of the RNA binds with the RNA, it begins to produce the the therapeutic protein within the patient, and when the tetracycline dosage is taken away or reduced, the RNA stops making the protein, enabling regulation and a consistent way to remain in the therapeutic window.

"The ability to control gene expression in mammalian cells is crucial for safe and efficacious gene therapies and for elucidating gene functions," the scientists write

"Although there are several gene regulation systems used in mammalian cells, none has been approved by the U.S. Food and Drug Administration for clinical applications, mainly because those systems use a regulatory protein that is foreign to the human body, which triggers an immune response against it," said Baylor medical research professor and the paper's principal investigator Laising Yen in a statement

"This strategy allows us to be more precise in the control of gene expression of a therapeutic protein. It enables us to adjust its production according to disease's stages or tune to the patients' specific needs, all using the FDA-approved tetracycline dose," Yen said