The Center for NeuroGenetics is focused on using molecular, genetic and clinical research to define the causes of neurodegenerative disease and to develop effective treatment strategies.
Genetic disorders that affect the nervous and muscular systems are responsible for a large number of devastating diseases including amyotrophic lateral sclerosis (ALS), Alzheimer’s disease, myotonic dystrophy (DM), Huntington disease (HD) and the spinocerebellar ataxias (SCAs). Each of these diseases progresses over a period of years and results in insidious decline and ultimately death. Although there has been a significant effort to understand and treat these disorders, progress has been slow. A major reason for the limited advancement in our understanding and treatment of these disorders is that we have not developed sufficiently integrated and multidisciplinary approaches to understand the causes and common pathways of these diseases.
The goal of the Center for NeuroGenetics is to advance our basic understanding of these disorders so we can develop rational therapeutic strategies for patients. Key aspects of our approach are to partner with affected families to identify novel disease genes and to link these patients with scientists working to understand these diseases using both clinical and basic science approaches.