From basic neuroscience to targeted treatments for ASD
The first step toward developing targeted biomedical interventions to address core symptoms of autism is to gain knowledge of the underlying biological processes that generate autistic behaviors. Several studies selected for funding provide this opportunity. For example, one of the most direct routes to uncovering the pathology associated with autism is to study brain tissue from individuals with ASD. Using post-mortem brain material, one of the studies will systematically investigate the affected brain networks, allowing identification of the cellular and molecular defects that characterize autism. Data from Neuropathology of the social-cognitive network in Autism: a comparison with other structural theories (Steven Chance, D.Phil, B.Sc., University of Oxford) will point the way toward biomedical treatments for autism.
Another approach to discovering the underlying biological mechanisms is to study the effects of autism risk genes on biochemical pathways, brain circuitry and behavior in animal models. For example, Functional study of synaptic scaffold protein SHANK3 and autism mouse model (Yong-Hui Jiang, M.D., Ph.D., Duke University) will examine the effects of mutations in the SHANK3 gene, which have been found in some individuals with autism. The gene creates a protein involved in synaptic function, disturbances of which are emerging as one of the central themes of autism pathophysiology. An additional mouse model study by Joshua Corbin, Ph.D., Children's Research Institute, Children's Hospital National Medica
|Contact: Jane E. Rubinstein|