Although gene therapy has shown much promise over the past decade, one of its major challenges continues to be controlling the expression of a transplanted gene once it has been delivered into a cell. As many scientists already have reported, transplanted genes may switch off prematurely, or, in some cases, they might not turn off fast enough, causing an undesirable overproduction of its replacement protein.
One way around this problem is to control the expression of the transplanted gene with a system controlled by a small molecule, for example rapamycin, a well-characterized immunosuppressive drug. As scientists have envisioned the strategy, they stitch a chemical switch next to the gene that only rapamycin (or derivative) molecules can control. Upon administration of the drug, the gene will turn on leading to protein production. Already, researchers have demonstrated the effectiveness of this approach in the liver, muscle, and eye.
Now, a team of scientists report in an article published online in the journal Gene Therapy they succeeded in getting the so-called "rapamycin gene-activation" system to work in the salivary glands. As the scientists noted, their finding could one day have important implications in treating a variety of oral and possibly systemic conditions with gene therapy.
National Institute of Dental and Craniofacial Research (NIDCR)
Message posted by: Rashmi Nemade
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