Scientists have created a new technique that allows them to “switch” genes on or off like a light—which could open the doors to more effective and safe gene therapy.
A team of researchers at Baylor College of Medicine published a study in the journal Nature Biotechnology that details how the technology allows them to regulate gene expression. This means that the switch tells genes to produce proteins, which can be used for everything from repairing broken tissue to performing key chemical reactions like energy creation.
This process also helps solve issues with traditional gene regulation methods. This process often requires introducing foreign proteins into human cells, which can be rejected by the body’s immune system.
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“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,” co-author Laising Yen, associate professor of pathology and immunology at Baylor, said in a statement.
The team created the switch by using molecules that interact with the body’s RNA. The switch can be turned on with an injection of an FDA-approved antibiotic called tetracycline. When this happens, the gene produces therapeutic proteins that can be used to treat diseases and conditions like autoimmune disorders, cancers, and infections.
The switch is variable too. If the patient requires a lot of proteins, doctors can dial it up by injecting more tetracycline. If they need a low, steady-stream of proteins, doctors can give a consistent low dose of the antibiotic.
“This strategy allows us to be more precise in the control of gene expression of a therapeutic protein,” Yen explained. “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.”
He later added that the technology isn’t just limited to one type of medical condition. “Our approach is not disease-specific, it can theoretically be used for regulating the expression of any protein, and potentially has many therapeutic applications.”
The technology is still a long way from being used in clinical settings. However, it paves the way for a near future where gene therapy becomes as easy as flipping a switch.
