King's College team IDs a gene target for Parkinson's

Parkinson's disease (PD) involves the progressive loss of motor function characterized by erratic muscle movement, as well as psychiatric symptoms and cognitive impairment. Treatment for PD remains limited and patients are left with therapies, like dopamine therapy, to address quality of life rather than prevent the disease progression.

Recently, researchers at King's College London (KCL) have unraveled a new gene associated with nerve function, a discovery that could eventually lead to treatment of those with neurodegenerative diseases like PD.

In a study published this week in the Proceedings of the National Academy of Sciences, it was found that a gene called HIFalpha regulates nerve signals in damaged mitochondria, and when it is genetically switched off, the activity and function of the nerve cells are restored.

Although this work was done in the fruit fly, HIFalpha is a gene conserved in higher order mammals including humans and therefore may be an important target for patients where damaged mitochondria has been reported--a common finding in neurodegenerative disease.

The benefit of such work may extend to diseases other than Parkinson's since the team successfully reproduced their findings in flies with Leigh syndrome, a rare neurological disease caused by a severe mitochondrial defect which occurs within the first year of a human life.

"Like their human counterparts flies with Parkinson's disease progressively lose motor function, which includes a negative impact on their ability to climb," said Dr. Joseph Bateman from the Institute of Psychiatry, Psychology & Neuroscience at KCL and a lead author of the study in a release. "Remarkably, we found that switching off a particular gene dramatically improved their motor function and climbing ability. The biggest surprise from our work is that damaged mitochondria produce a signal that actively prevents nerve cells from working properly."

Claire Bale, head of research communications at Parkinson's UK, added, "This discovery adds a new piece to the intricate jigsaw puzzle of genetic factors that play a part in Parkinson's."

- here's the release

Suggested Articles

Removing the IRE1-alpha gene from beta cells in mouse models of Type 1 diabetes restored normal insulin production, scientists found.

Selectively targeting TGF-beta1 with Scholar Rock's SRK-181 overcame primary resistance to checkpoint inhibitor therapy in mice.

Enhertu produced a 55.6% objective response rate in HER2-positive non-small cell lung cancer patients in a phase 1 trial.