University of Iowa News
April 12, 2006
UI Neuroscientist Receives Grant To Study Potential Dystonia Therapy
Pedro Gonzalez-Alegre, M.D., assistant professor of neurology in the University of Iowa Roy J. and Lucille A. Carver College of Medicine, has received a $200,000 grant from the Dystonia Medical Research Foundation to study therapeutic RNA interference for DYT1 dystonia.
Dystonias are a group of disabling movement disorders that affect 300,000 people in North America. DYT1 dystonia, the most common inherited dystonia, is caused by a genetic mutation that produces an abnormal version of a protein called torsinA.
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.
Abnormal torsinA protein stops certain brain cells from working, but it does not destroy the cells, which means that suppressing the toxic protein may produce a complete cure for the disease.
RNA interference, or RNAi, holds promise as a therapy for many diseases, including DYT1, because it can selectively suppress or silence disease-causing genes.
Gonzalez-Alegre and colleagues have previously shown that RNAi can specifically suppress abnormal torsinA in a human neural cell model of DYT1. The RNAi treatment appears to rescue the cells and return them to a normal state. Using the DMRF grant to extend his studies, Gonzales-Alegre will test the safety and efficacy of this potential therapy in an animal model of DYT1. In particular, he will examine whether the treatment is effective in improving or even reversing the movement problems in animals with the DYT1 mutation.
For more information on dystonia and the Dystonia Medical Research Foundation visit www.dystonia-foundation.org.
University of Iowa Health Care describes the partnership between the UI Roy J. and Lucille A. Carver College of Medicine and UI Hospitals and Clinics and the patient care, medical education and research programs and services they provide. Visit UI Health Care online at www.uihealthcare.com.
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