Gene therapy protects neurons in Parkinson's disease mouse study

A group of scientists has concluded that a noninheritable form of Parkinson's disease (PD) may be caused by functional changes in the immune regulating gene interferon-beta (IFNβ). And correction of IFNβ with gene therapy can prevent neuronal death and disease progression in a mouse model of PD.

The scientists, based at the University of Copenhagen, Denmark, and led by professor Shohreh Issazadeh-Navikas, published their work in Cell earlier this week.

"IFNβ is essential for neurons ability to recycle waste proteins. Without this, the waste proteins accumulate in disease-associated structures called Lewy bodies and with time the neurons die," said Patrick Ejlerskov, first author and an assistant professor at the university.

With the function of IFNβ absent, they observed similar outcomes to PD in a mouse model where they genetically deleted it. PD is well known for its hereditary gene mutations that cause familial Parkinson's. What is less known is "nonfamilial PD," which overwhelmingly causes the bulk of all PD cases in humans.

"This is one of the first genes found to cause pathology and clinical features of non-familial PD and dementia with Lewy bodies, through accumulation of disease-causing proteins," Issazadeh-Navikas said. "Our hope is that this knowledge will enable development of more effective treatment of PD."

- here's the release
- get the research abstract

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