Pathogenesis and Treatment of Huntington's Disease

There is currently no effective treatment for Huntington's disease, a progressive and ultimately fatal neurological disorder caused by a defect in the Huntington Disease gene. Symptoms of the inherited disease, which usually appear at mid-life, include abnormalities in movement, difficulties with awareness and judgement, and emotional instability. Using genetically altered mice, Jeremy Van Raamsdonk is investigating the underlying genetic and cellular changes that give rise to Huntington's disease and potential treatment strategies. The research involves testing both drug and gene-based treatments targeted at the root cause of the disease, as well as assessing treatments to minimize the damage to the nervous system. By developing specific treatment strategies, Van Raamsdonk aims to limit damage to nervous system cells and increase the lifespan and quality of life for people with Huntington's disease.