Huntington's Disease Clinical Research

Huntington's Disease Clinical Research

Huntington's disease is a genetic neurological disorder characterized after onset by uncoordinated, jerky body movements and a decline in some mental abilities for which there is no present cure or effective treatments.

Research into the mechanism of Huntington's disease (HD) has focused on identifying the functioning of the huntingtin protein (Htt), how mutant huntingtin (mHtt) differs or interferes with it, and the brain pathology that the disease produces. Research is conducted using in vitro methods, animal models and human volunteers. Animal models are critical for understanding the fundamental mechanisms causing the disease and for supporting the early stages of drug development. The identification of the causative gene has enabled the development of many transgenic animal models including nematode worms, Drosophila fruit flies, mice, rats, sheep, pigs and monkeys that express mutant huntingtin and develop progressive neurodegeneration and HD-like symptoms.

Three broad approaches are under study to attempt to slow the progression of Huntington's disease: reducing production of the mutant protein, improving cells' ability to survive its diverse harmful effects, and replacing lost neurons.