Huntington's Disease - Research Directions

Research Directions

Research into the mechanism of HD has focused on identifying the functioning of Htt, how 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. Animals with chemically induced brain injury exhibit HD-like symptoms and were initially used, but they did not mimic the progressive features of the disease. 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.