AANS Neurosurgeon | Volume 26, Number 4, 2017

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In Huntington's Disease, Traffic Jams in the Cell's Control Center Kill Brain Cells

Working with mouse, fly and human cells and tissue, Johns Hopkins researchers report new evidence that disruptions in the movement of cellular materials in and out of a cell’s control center — the nucleus — appear to be a direct cause of brain cell death in Huntington’s disease, an inherited adult neurodegenerative disorder. Moreover, they suggest, laboratory experiments with drugs designed to clear up these cellular “traffic jams” restored normal transport in and out of the nucleus and saved the cells. The researchers also conclude that potential treatments targeting the transport disruptions they identified in Huntington’s disease neurons may also work for other neurodegenerative diseases, such as ALS and forms of dementia.

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