Potential Therapeutic Target for Huntington's Disease Identified
Researchers studying yeast cells have identified a metabolic enzyme as a potential therapeutic target for treating Huntington's disease, a fatal inherited neurodegenerative disorder that affects an estimated 30,000 people in the United States.
Paul Muchowski, assistant professor of pharmacology, led the group of scientists from the University of Washington School of Medicine and the University of Maryland School of Medicine in Baltimore.
The researchers performed an experiment known as a loss-of-function suppressor screen, which searches for genes that, when switched off, reduce the toxic effects of the mutant protein associated with Huntington's. One of the genes identified encodes an enzyme, called KMO, that has been previously implicated in the disease. The enzyme functions in a metabolic pathway that is activated at early stages of the disease in people with Huntington's, as well as in animal models of the disease.
Muchowski says the good news is that there is a chemical compound available that inhibits KMO activity. Researchers are testing the compound in a mouse model of Huntington's disease.
The research findings appear in the May issue of Nature Genetics, and can be found online at the Nature Genetics Web site, http://www.nature.com/ng/index.html