Huntington’s Disease claimed the life of Woody Guthrie and many other Americans. Now Iowans are on the track of a treatment that may slow, if not eradicate, the degenerative disease. Doctor Beverly Davidson is the Roy J. Carver chair in Internal Medicine at the University of Iowa Medical School. She says a gene has mutated, in people who develop the disease, and it produces a toxic form of one of the body’s vital proteins. Her researchers tried to find ways to “turn off” or silence the mutant protein, using a technique called R-N-A interference. In their studies, they tested that tactic in several ways. The scientists tested to see if their RNA interference helped keep Huntington’s-affected mice from developing typical symptoms, by testing whether they suffered the typical loss of abilities like wavering when they walk or becoming unable to keep their balance on a rotating rod. Another thing they did was test levels of the “mutant protein” in brain cells, and they found their therapy cut the toxic protein to 40-percent of the level at which it’s found in Huntington’s patients. The “mutant Huntington” protein also tends to form characteristic clumps in cells, which were reduced in subjects given the RNA-interference therapy. Dr. Davidson says the evidence that both behavior and cell appearance improved could be very encouraging news for the use of this therapy that cuts the toxic protein level. The data suggests they don’t have to “turn it off” all the way to have some very beneficial effects, which she says is exciting. The U of I researchers will do more mouse studies for now, using subjects bred to be more and more similar to human patients, before they begin studies of the treatment in human volunteers.
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