January 18, 2018
A team of NIH scientists and collaborators has uncovered a new potential strategy against Huntington’s disease, an inherited, fatal neurodegenerative disorder that has no cure. In a recently published study, researchers at NIH’s National Center for Advancing Translational Sciences (NCATS), the University of Michigan and Baylor College of Medicine demonstrated that blocking the activity of an enzyme called PIP4Kγ reduced the effects of the disease in both cell and animal models. The findings indicate that inhibiting the enzyme triggers a normal-cell-“recycling” system that helps lessen the buildup of malformed, toxic proteins in brain cells that is associated with neurological problems.
Juvenile Huntington disease