The researchers said a single gene mutation kills two types of brain cells in Huntington's patients and were able to use a unique approach to switch that gene off, in laboratory studies involving mice.
 

The research, published in the journal Nature Medicine, sheds new light on where Huntington's starts in the brain and suggests new targets and routes for therapeutic drugs to slow the devastating disease, which strikes an estimated 35,000 Americans, Medical Xpress reports.

"From day one of conception, the mutant gene that causes Huntington's appears everywhere in the body, including every cell in the brain," explained X. William Yang, professor of psychiatry and biobehavioral sciences at the Semel Institute for Neuroscience and Human Behavior at the University of California-Los Angeles. "Before we can develop effective strategies to treat the disorder, we need to first identify where it starts and how it ravages the brain."
 

Huntington's disease is passed from parent to child through a mutation in a gene called huntingtin that eventually causes brain atrophy that deprives patients of their ability to move, speak, eat, and think clearly. There is no cure and people with aggressive cases may die in as little as 10 years after diagnosis.
 

In the new study, Yang developed a "genetic scissors" to shut down the defective gene.
 

"Our research helps to shed lights on an age-old question in the field," Yang said. "Where does Huntington's disease start? Equally important, our findings provide crucial insights on where to target therapies to reduce mutant gene levels in the brain."