|Project Title||Principal Investigator||Institution|
|Investigation of the role of MET kinase in autism||Dawson, Ted||Johns Hopkins University School of Medicine|
|Control of synaptic protein synthesis in the pathogenesis and therapy of autism||Kelleher, Raymond||Massachusetts General Hospital|
|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|
|Mice lacking Shank postsynaptic scaffolds as an animal model of autism||Sheng, Morgan||Massachusetts Institute of Technology|
|Neural and cognitive mechanisms of autism||Sur, Mriganka||Massachusetts Institute of Technology|
|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|
|Using Drosophila to model the synaptic function of the autism-linked NHE9||Littleton, J. Troy||Massachusetts Institute of Technology|
|Genetic models of serotonin transporter regulation linked to mental disorders||Ramamoorthy, Sammanda||Medical University of South Carolina|
|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|
|Animal models of neuropsychiatric disorders||Crawley, Jacqueline||National Institutes of Health|
|Regulation of gene expression in the brain||Young, Walter||National Institutes of Health|
|Synaptic plasticity, memory and social behavior||Chevere-Torres, Itzamarie||New York University|
|Methods for production of ICSI and SCNT derived macaque stem cells||Schatten, Gerald||Southwest Foundation For Biomedical Research|
|Optimization of methods for production of both ICSI- and SCNT derived baboon embryonic stem cells||Schatten, Gerald||Southwest Foundation For Biomedical Research|
|Micro-RNA regulation in pluripotent stem cells||Schatten, Gerald||Southwest Foundation 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|
|Exploring the neuronal phenotype of autism spectrum disorders using induced pluripotent stem cells||Hallmayer, Joachim; Dolmetsch, Ricardo||Stanford University|
|Synaptic deficits of iPS cell-derived neurons from patients with autism||Mao, Rong||Stanford University|
|Function and dysfunction of neuroligins||Sudhof, Thomas||Stanford University|
|Identification of autism genes that regulate synaptic Nrx/Nlg signaling complexes||Garner, Craig||Stanford University|
|Role of L-type calcium channels in hippocampal neuronal network activity||Owen, Scott||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.