Researchers at the University of Iowa in Iowa City this month made public the results of their research into stopping dominant progressives brain disorders in mice.
A dominant progressive brain disorder is a disease caused when an infant inherits one copy of a mutant gene which results in the production of mutant proteins that cause loss of cognitive abilities. The most widely occurring form of this sort of disease in the United States is Huntington’s disease, with as many as 250,000 sufferers in this country alone. Huntington’s disease usually manifests itself in a victim’s mid-to-late 30s and sufferers typically live only 15-20 years after diagnosis while suffering progressively worsening brain degeneration.
The University of Iowa researchers used gene therapy to treat mice afflicted spioncerebellar ataxia type 1. The disease produces a mutated protein in the brain that eventually leads the mice to have difficulty in walking. Like Huntington’s it is not a result of a missing gene — which gene therapy has traditionally been used to treat in animal models and in a small number of humans — but rather a mutant gene inherited from a parent.
Nonetheless, researchers used a virus to carry modified genetic material to the mice. The genetic material was designed to bind with and block the expression the defective gene. After the mice were injected with the virus, the production of the proteins causing the disease stopped and the mice appeared to improve their ability to walk.
The researchers then took the process one step further and used the same method to see if genetic material could be effective when added in vitro to human Huntington’s cell cultures. After exposing the cell cultures to a different virus containing the genetic materials, the cells stopped producing the proteins that cause Huntington’s disease.
Lead researcher Beverley Davidson said the she hopes this discovery moves quickly into clinical trials to see if it can be effective in treating Huntington’s in human beings,
The data are very promising; we hope we will be able to use RNA interference as a therapy for dominant neurodegenerative diseases.
Cure hoped for Huntington’s sufferers. Erika Chek, Nature, June 9, 2004.
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