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| A microglia cell (shown in green) and corticostriatal synapses (purple) from a patient with Huntington’s disease. Image Credit: Dan Wilton |
A new study led by researchers at Boston Children’s Hospital and Harvard Medical School reveals how the process of Huntington’s disease begins well before symptoms appear — and shows that in mice, the process can be blocked to prevent cognitive problems related to Huntington’s.
If the findings hold true in humans, they raise the possibility of intervening early in the disease in people who carry the Huntington’s gene mutation.
The work, published in Nature Medicine, also could shed light on other neurodegenerative disorders.
The team found in patient tissue samples and mouse models that two players in the immune system — complement proteins and microglia — are activated very early in Huntington’s, leading to loss of synapses in the brain before cognitive and motor symptoms emerge. The researchers revealed how and where the synapses are lost.
The findings corroborate a potential treatment that’s currently in clinical trials for the disease.
The study was led by senior author Beth Stevens, HMS associate professor of neurology at Boston Children’s, and first author Dan Wilton, HMS research fellow in neurology in the Stevens lab.

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