PACEMAKER: Spring 2006

Neurosciences Team Uses Gene Silencing to Fix Movement Disorder

Jennifer Brown

Researchers at the University of Iowa Roy J. and Lucille A. Carver College of Medicine continue to advance a potential new treatment for neurological disorders. RNA interference, or RNAi, holds promise as a therapy for many diseases, including brain disorders, because it can selectively suppress or silence disease-causing genes.

In a new study published in the Nov. 9 issue of the Journal of Neuroscience, Pedro Gonzalez-Alegre, M.D., UI assistant professor of neurology, and his colleagues use RNAi to restore human neural cells to normal by selectively silencing a gene that causes the movement disorder DYT1 dystonia.

The findings may also help researchers apply RNAi to other brain diseases such as Huntington's disease.

Starting in childhood, patients with DYT1 dystonia develop involuntary muscle contractions that cause twisting of limbs. The disease progresses over several years to involve the whole body and is very physically disabling, although patients have normal brain function. There currently is no cure and only limited treatment for DYT1 dystonia which, although rare, is the most common inherited dystonia.

The study was funded by the National Institute of Neurological Disorders and Stroke and the Dystonia Medical Research Foundation.