Gene Therapy Injected Into The Brains' Of Mice With Huntington's Disease

Source: Rush University Medical Center
Posted: June 12, 2006

Researchers at Rush University Medical Center, Chicago, and Ceregene Inc., San Diego, have successfully used gene therapy to preserve motor function and stop the anatomic, cellular changes that occur in the brains of mice with Huntington’s disease (HD). This is the first study to demonstrate that, using this delivery method, symptom onset might be prevented in HD mice with this treatment.

“This could be an important step toward a disease modifying therapy,” says co-author Jeffrey H. Kordower, Ph.D., director of the Research Center for Brain Repair at Rush. “We could potentially be stopping the disease process in its tracks, delaying symptoms from ever showing up.”

Commentary: Hopefully this finding will lead to clinical applications and successful treatments for humans with Huntington's Disease.