Research into the mechanism of HD is 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, genetically modified animals, (also called transgenic 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 genetically modified organisms including nematodes (roundworms), Drosophila fruit flies, and genetically modified mammals including mice, rats, sheep, pigs and monkeys that express mutant huntingtin and develop progressive neurodegeneration and HD-like symptoms.
Research into the mechanism of HD is 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 genetically modified animals (also called transgenic 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 genetically modified organisms including nematodes (roundworms) Drosophila fruit flies and genetically modified mammals including mice rats sheep pigs and monkeys that express mutant huntingtin and develop progressive neurodegeneration and HD-like symptoms