A Novel Therapy for Huntington's Disease.

In 1979, while at the National Institutes of Health, now Columbia University professor Nancy Wexler and colleagues traveled to Venezuela to study the world's largest family with Huntington's disease. That led to identifying the disease gene at the tip of human chromosome 4 and the race to find a drug that can treat people who carry the fatal gene prior to the onset of symptoms. Our author believes that a new strategy tied to turning off targeted genes could have profound implications for therapy development for Huntington's and other neurodegenerative diseases.