BioCentury
ARTICLE | Cover Story

Low-Rhes approach to Huntington's

June 25, 2009 7:00 AM UTC

A central mystery of Huntington's disease has been why the mutant form of the huntingtin protein responsible for the condition is found throughout the body but only causes pathology in the brain's corpus striatum. Researchers at The Johns Hopkins University School of Medicine may have the answer-a protein called Rhes that is specific to the corpus striatum and mediates the neurotoxic effects of mutant huntingtin protein.1

The target looks to be druggable, thus potentially expanding the current target landscape in HD (see "Huntington's disease pipeline"), and could be explored using adaptations to existing animal models...