AANS Neurosurgeon | Volume 26, Number 3, 2017

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Basis of ‘Leaky’ Brain Blood Vessels in Huntington’s Disease Identified

UCI-led stem cell study points to new treatments for this fatal disorder

By using induced pluripotent stem cells to create endothelial cells that line blood vessels in the brain for the first time for a neurodegenerative disease, University of California, Irvine neurobiologists and colleagues have learned why Huntington’s disease patients have defects in the blood-brain barrier that contribute to the symptoms of this fatal disorder. “Now we know there are internal problems with blood vessels in the brain,” said study leader Leslie Thompson, UCI professor of psychiatry & human behavior and neurobiology & behavior. “This discovery can be used for possible future treatments to seal the leaky blood vessels themselves and to evaluate drug delivery to patients with HD.” The blood-brain barrier protects the brain from harmful molecules and proteins. It has been established that in Huntington’s and other neurodegenerative diseases there are defects in this barrier adding to HD symptoms. What was not known was whether these defects come from the cells that constitute the barrier or are secondary effects from other brain cells. 

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