|Project Title||Principal Investigator||Institution|
|Vasopressin receptors and social attachment||Young, Larry||Emory University|
|Using zebrafish and chemical screening to define function of autism genes||Sive, Hazel||Whitehead Institute for Biomedical Research|
|Using iPS cells to study genetically defined forms with autism||Dolmetsch, Ricardo||Stanford University|
|Using induced pluripotent stem cells to identify cellular phenotypes of autism||Dolmetsch, Ricardo||Stanford University|
|Using Drosophila to model the synaptic function of the autism-linked NHE9||Littleton, J. Troy||Massachusetts Institute of Technology|
|Transgenic mouse model to address heterogeneity in autism spectrum disorders||Blakely, Randy||Vanderbilt University|
|The role of SHANK3 in the etiology of autism spectrum disorder||Bangash, M. Ali||Johns Hopkins University|
|The role of SHANK3 in autism spectrum disorders||Buxbaum, Joseph||Mount Sinai School of Medicine|
|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|
|Systematic analysis of neural circuitry in mouse models of autism||Osten, Pavel||Cold Spring Harbor Laboratory|
|Synaptic plasticity, memory and social behavior||Chevere-Torres, Itzamarie||New York University|
|Synaptic deficits of iPS cell-derived neurons from patients with autism||Mao, Rong||Stanford University|
|Synaptic and circuitry mechanisms of repetitive behaviors in autism||Feng, Guoping||Massachusetts Institute of Technology|
|Small-molecule compounds for treating autism spectrum disorders||Philpot, Ben||The University of North Carolina at Chapel Hill|
|Shank3 mutant characterization in vivo||Kouser, Mehreen||University of Texas Southwestern Medical Center|
|Serotonin, corpus callosum, and autism||Lin, Rick||University of Mississippi Medical Center|
|Serotonin, autism, and investigating cell types for CNS disorders||Dougherty, Joseph||The Rockefeller University|
|Role of UBE3A in neocortical plasticity and function||Ehlers, Michael||Duke University|
|Role of L-type calcium channels in hippocampal neuronal network activity||Owen, Scott||Stanford University|
|Role of a novel Wnt pathway in autism spectrum disorders||Reichardt, Louis||University of California, San Francisco|
|Regulation of gene expression in the brain||Young, Walter||National Institutes of Health|
|Preclinical testing of novel oxytocin receptor activators in models of autism phenotypes||Pedersen, Cort||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|
|Preclinical testing of novel oxytocin receptor activators in models of autism phenotypes||Moy, Sheryl||University of North Carolina at Chapel Hill|
|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.