New research could lead to advanced treatments for potentially deadly diseases. Researchers with University of Iowa Health Care have discovered a way to selectively silence a diseased gene without affecting the normal gene. The breakthrough could eventually lead to new treatments for cancer and other disorders.
University of Iowa researchers used new technology known as RNA interference to selectively silence a mutant gene that causes a neuro-degenerative condition called Machado-Joseph disease. At the same time, researchers were able to leave the normal gene alone. Henry Paulson, M.D., a University of Iowa Health Care neurologist, led the study.
"What we did is we targeted those mutations using this RNA interference technology," says Paulson. "Were able to silence expression of the mutant gene but keep the normal gene going."
Turning off a mutant gene while keeping the normal gene active could be useful in treating certain inherited diseases, including cancer and Huntingtons disease.
"At this point we dont have a cure or even a partially preventive therapy for either of these diseases, or I might add, for almost all neuro-degenerative diseases. Our hope is that this kind of technology will be brought to bear in developing therapies for these diseases," says Paulson.
The same type of genetic mutation causes a number of other neuro-degenerative disorders, including Alzheimers and Parkinsons disease. Paulson says further testing is needed, but he believes this is the next step toward potential treatments.
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Henry Paulson, M.D.
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