U-of-I study finds way to silence mutant genes

Genes come in pairs, one from your mom and one from your dad. Inheriting one bad, or mutant, gene from either parent may cause disease. A University of Iowa study shows it’s possible to silence that mutant gene without affecting the normal gene. The study’s lead author Victor Miller, a graduate student at the U-of-I, says they focused on diseases where there’s a single bad copy from the mother or father that leads to a neurological disorder. The findings show the gene-silencing technique may be useful in treating cancer, Huntington’s disease and similar genetic disorders and viral diseases. Miller says “What we’ve done is use special small molecules to attempt to turn off the bad gene and thus hopefully prevent you from ever developing the disease but leave the one good copy you have intact to perform the normal function it would otherwise do in your body.” He says the study asserts it’s possible that diseases could be prevented from developing if caught early in children, though it’s also possible diseased adults could be helped. Miller says “We hope that even if you already have symptoms, that turning off more production of that protein will either help you improve or at least will prevent you from getting any worse.” Miller says the research team focused as a starting point on rare genetic diseases. He says these rare diseases have a lot in common with things like Alzheimer’s, Parkinson’s, and Huntington’s Disease which effect many more people. The study is appearing this afternoon in the Online Early Edition of the Proceedings of the National Academy of Sciences (www.pnas.org).