|Project Title||Principal Investigator||Institution|
|Neurobiological mechanism of 15q11-13 duplication autism spectrum disorder||Anderson, Matthew||Beth Israel Deaconess Medical Center|
|The role of SHANK3 in the etiology of autism spectrum disorder||Bangash, M. Ali||Johns Hopkins University|
|Neurobiology of mouse models for human chr 16p11.2 microdeletion and fragile X||Bear, Mark||Massachusetts Institute of Technology|
|Development of a high-content neuronal assay to screen therapeutics for the treatment of cognitive dysfunction in autism spectrum disorders||Bear, Mark||Massachusetts Institute of Technology|
|Transgenic mouse model to address heterogeneity in autism spectrum disorders||Blakely, Randy||Vanderbilt University|
|The genetic control of social behavior in the mouse||Blanchard, Robert||University of Hawai'i at Manoa|
|Neurobiology of sociability in a mouse model system relevant to autism||Brodkin, Edward||University of Pennsylvania|
|Identifying genetic modifiers of rett syndrome in the mouse||Buchovecky, Christine||Baylor College of Medicine|
|The role of SHANK3 in autism spectrum disorders||Buxbaum, Joseph||Mount Sinai School of Medicine|
|A preclinical model for determining the role of AVPR1A in autism spectrum disorders||Charles, Rhonda||Mount Sinai School of Medicine|
|Synaptic plasticity, memory and social behavior||Chevere-Torres, Itzamarie||New York University|
|Animal models of neuropsychiatric disorders||Crawley, Jacqueline||National Institutes of Health|
|Investigation of the role of MET kinase in autism||Dawson, Ted||Johns Hopkins University School of Medicine|
|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|
|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|
|Patient iPS cells with copy number variations to model neuropsychiatric disorders||Ellis, James||The Hospital for Sick Children|
|Dissecting the circuitry basis of autistic-like behaviors in mice||Feng, Guoping||Massachusetts Institute of Technology|
|Synaptic and circuitry mechanisms of repetitive behaviors in autism||Feng, Guoping||Massachusetts Institute of Technology|
|Functional genomic dissection of language-related disorders||Fisher, Simon||University of Oxford|
|Deriving neuroprogenitor cells from peripheral blood of individuals with autism||Fujinami, Robert||University of Utah|
|Identification of autism genes that regulate synaptic Nrx/Nlg signaling complexes||Garner, Craig||Stanford University|
|Genomic imbalances at the 22q11 locus and predisposition to autism||Gogos, Joseph||Columbia University|
|Exploring the neuronal phenotype of autism spectrum disorders using induced pluripotent stem cells||Hallmayer, Joachim; Dolmetsch, Ricardo||Stanford University|
|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.