Origin of Huntington's disease found in brain; insights to help deliver therapy
The gene mutation that causes Huntington's disease appears in every cell in the body, yet kills only two types of brain cells. Why? UCLA scientists used a unique approach to switch the gene off in individual brain regions and zero in on those that play a role in causing the disease in mice.
Published in the April 28 online edition of Nature Medicine, the research sheds light on where Huntington's starts in the brain. It also suggests new targets and routes for therapeutic drugs to slow the devastating disease, which strikes an estimated 35,000 Americans.
"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 UCLA. "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. Scientists blame a genetic "stutter" -- a repetitive stretch of DNA at one end of the altered gene -- for the cell death and brain atrophy that progressively deprives patients of their ability to move, speak, eat and think clearly. No cure exists, and people with aggressive cases may die in as little as 10 years.
more
http://www.sciencedaily.com/releases/2014/04/140428120654.htm
