IOWA CITY, Iowa, June 15 (UPI) -- U.S. researchers have found gene therapy may be used to treat neurodegenerative disorders such as Huntington's disease, for which there is no cure.
Neurodegenerative diseases are caused when the brain accumulates mutant proteins. In Huntington's disease, these proteins worsen symptoms that include dementia, uncontrolled muscle movement, deteriorating cognitive processes and personality disintegration.
Researchers at the University of Iowa injected corrective DNA and RNA sequences into mice that had a disease that mimics spinocerebellar ataxia type 1, which eventually leaves patients unable to walk.
They found the injections eliminated pockets of damaged brain tissue in the mice and corrected the physical symptoms of the disorder.
This is the first time gene therapy has been shown to cure such a disease, the researchers said, adding it also could be the first cure for dominant progressive brain disorders, where patients inherit a defective copy of a gene from one parent.
The researchers also discovered that RNA interference -- a technique used to isolate pieces of genetic material that bind to a mutant gene -- stopped human cells in culture dishes from forming the mutant proteins found in Huntington's disease.
