Gene therapy prevents Huntington-like disease in mice

In neurodegenerative disorders such as Huntington's disease and spinocerebellar ataxia, a single mutated gene produces a protein that is toxic to cells. Researchers at the University of Iowa, using gene therapy in the brains of living mice with a similar type of neurodegenerative disease, were able to "silence" the defective gene. The mice treated with the process, called RNA interference, had normal movement and coordination, and their brain cells were protected from the destruction normally caused by the disease. In addition, the RNA interference itself did not appear to damage normal brain cells. The researchers are optimistic about using this type of gene therapy to treat these types of neurodegenerative disorders in humans.