Huntington’s disease (HD) is a devastating, inherited neurological disorder that causes psychiatric, cognitive and movement deficits.The Thompson laboratory is actively engaged in investigating the fundamental molecular and cellular events that underlie how the mutant HD gene causes degeneration of specific brain cell populations to induce motor and cognitive decline and premature death of patients with the ultimate goal to develop new therapeutic approaches. Several potential drug targets that have emerged are in various phases of target validation and preclinical and clinical trials, including stem cell-based therapies. The laboratory also focuses on understanding casual mechanisms that underlie HD and Amyotrophic Lateral Sclerosis and more recently X-linked Dystonia-Parkinsonism with the goal of developing treatments for the disease.  The research benefits from the integrated use of patient iPSCs and mouse models of disease together with the studies of RNA biology and network-based bioinformatics.