Genes come in pairs, one copy from your mom and one from your dad. In some genetic conditions, inheriting one bad, or mutant, gene copy from either parent is sufficient to cause disease. University of Iowa researchers have shown that it is possible to silence a mutant gene without affecting expression of the normal gene.
The findings suggest that the gene-silencing technique might one day be useful in treating many human diseases, including cancer, Huntington’s disease and similar genetic disorders, and viral diseases, where it would be desirable to selectively turn off certain genes that cause problems.
In particular, the UI researchers were able to silence mutant genes without affecting the normal gene copy even when the mutant and the normal gene differ by as little as a single letter in the genetic code. The study will appear this week in the Online Early Edition of the Proceedings of the National Academy of Sciences (www.pnas.org).
Jennifer Brown | University of Iowa
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