|Project Title||Principal Investigator||Institution|
|Patient iPS cells with copy number variations to model neuropsychiatric disorders||Ellis, James||The Hospital for Sick Children|
|Serotonin, autism, and investigating cell types for CNS disorders||Dougherty, Joseph||The Rockefeller University|
|Autism iPSCs for studying function and dysfunction in human neural development||Loring, Jeanne||The Scripps Research Institute|
|Small-molecule compounds for treating autism spectrum disorders||Philpot, Ben||The University of North Carolina at Chapel Hill|
|A novel cell-based assay for autism research and drug discovery||Restifo, Linda||University of Arizona|
|Cntnap2 in a behavioral model of autism||White, Stephanie||University of California, Los Angeles|
|Basal ganglia circuitry and molecules in pathogenesis of motor stereotypy||Yang, Xiangdong||University of California, Los Angeles|
|Neurogenomics in a model for procedural learning||Hilliard, Austin||University of California, Los Angeles|
|High content screens of neuronal development for autism research||Halpain, Shelley||University of California, San Diego|
|Insight into MeCP2 function raises therapeutic possibilities for Rett syndrome||Lomvardas, Stavros||University of California, San Francisco|
|Dissecting the neural control of social attachment||Shah, Nirao||University of California, San Francisco|
|Role of a novel Wnt pathway in autism spectrum disorders||Reichardt, Louis||University of California, San Francisco|
|The genetics of restricted, repetitive behavior: An inbred mouse model||Lewis, Mark||University of Florida|
|The genetic control of social behavior in the mouse||Blanchard, Robert||University of Hawai'i at Manoa|
|A mouse knock-in model for ENGRAILED 2 autism susceptibility||Millonig, James||University of Medicine & Dentistry of New Jersey|
|Serotonin, corpus callosum, and autism||Lin, Rick||University of Mississippi Medical Center|
|Preclinical testing of novel oxytocin receptor activators in models of autism phenotypes||Pedersen, Cort||University of North Carolina at Chapel Hill|
|Novel strategies to manipulate Ube3a expression for the treatment of autism and Angelman syndrome||Roth, Bryan||University of North Carolina at Chapel Hill|
|Preclinical testing of novel oxytocin receptor activators in models of autism phenotypes||Jarstfer, Michael||University of North Carolina at Chapel Hill|
|NrCAM, a candidate susceptibility gene for visual processing deficits in autism||Maness, Patricia||University of North Carolina at Chapel Hill|
|Preclinical testing of novel oxytocin receptor activators in models of autism phenotypes||Moy, Sheryl||University of North Carolina at Chapel Hill|
|Characterization of a novel mouse model of restricted repetitive behaviors||Moy, Sheryl||University of North Carolina at Chapel Hill|
|Neuropharmacology of motivation and reinforcement in mouse models of autistic spectrum disorders||Malanga, C.J.||University of North Carolina School of Medicine|
|Functional genomic dissection of language-related disorders||Fisher, Simon||University of Oxford|
|Neurobiology of sociability in a mouse model system relevant to autism||Brodkin, Edward||University of Pennsylvania|
|IACC Strategic Plan Objective||2008||2009||2010||2011||2012||Total|
|Standardize and validate at least 20 model systems (e.g., cellular and/or animal) that replicate features of ASD and will allow identification of specific molecular targets or neural circuits amenable to existing or new interventions by 2012.
IACC Recommended Budget: $75,000,000 over 5 years
|4.S.B. Funding: The recommended budget was met. Significantly more than the recommended minimum budget was allocated to projects specific to this objective.
Progress: More than 90 projects were supported to develop animal models.
Remaining Gaps, Needs, and Opportunities: Planning Group members discussed whether the amount of investment in this area is appropriate when compared to investments in clinical trials and other later stage studies. Invited experts suggested that the current stage of scientific research in ASD requires pre-clinical research to identify targets from animal and cellular models. Similar to cancer treatment development pathways, which spanned 20-30 years, research in ASD must invest in model systems to understand the fundamental biology from which translation to the clinic can be built. The translational validity of research in non-human animals cannot be determined until human trials are conducted, thus the need for rapid progress to clinical studies in humans is important.