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Basic Science Research Award 20151
MRI showing cerebellar atrophy in ataxia patients3
The Center for NeuroGenetics (CNG) will integrate molecular, genetic and clinical approaches to define the causes of neurodegenerative disease and develop effective treatment strategies.
Impact and Burden of Neurological and Neuromuscular Disease
Genetic disorders that affect the nervous and muscular systems are responsible for a large number of devastating diseases including amyotrophic lateral sclerosis (ALS), muscular dystrophy (DM), spinal muscular atrophy (SMA), 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 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 talented faculty working to understand these diseases using both clinical and basic science approaches.