UCI researchers make Huntington’s breakthrough

Researchers at UC Irvine and MIT have made a significant breakthrough

in the fight against Huntington’s disease, a disorder of the nervous

system.

The researchers have identified a man-made protein that halts the

progression of the disease in fruit flies, which may contribute to

finding effective ways to use gene therapy to prevent or stop the disease

from spreading.

Leslie Thompson, assistant professor of psychiatry in the College of

Medicine, and Larry Marsh, professor of developmental biology in the

School of Biological Sciences, joined two MIT researchers in creating a

protein that binds tightly to another mutated protein that is produced by

the genetic changes that cause Huntington’s disease.

While gene therapy would be one means for this protein to successfully

combat the disease, scientists have encountered a number of obstacles to

gene therapy.

“This study helps focus on finding synthetic drugs or other chemicals

that can disrupt the accumulation of the [mutated protein] and may be

readily administered using more traditional medical techniques, instead

of gene therapy,” Marsh said. “We have some reason to hope that such

chemicals may halt the disease’s progress.”

The study is published in this month’s issue of Nature Genetics.