|Project Title||Principal Investigator||Institution|
|Interaction between MEF2 and MECP2 in the pathogenesis of autism spectrum disorders -2||Nakanishi, Nobuki||Burnham Institute|
|Systematic analysis of neural circuitry in mouse models of autism||Osten, Pavel||Cold Spring Harbor Laboratory|
|Role of L-type calcium channels in hippocampal neuronal network activity||Owen, Scott||Stanford University|
|Novel probiotic therapies for autism||Patterson, Paul||California Institute of Technology|
|Preclinical testing of novel oxytocin receptor activators in models of autism phenotypes||Pedersen, Cort||University of North Carolina at Chapel Hill|
|Small-molecule compounds for treating autism spectrum disorders||Philpot, Ben||The University of North Carolina at Chapel Hill|
|Neurogenetic model of social behavior heterogeneity in autism spectrum disorders||Platt, Michael||Duke University|
|High-resolution diffusion tensor imaging in mouse models relevant to autism||Poptani, Harish||University of Pennsylvania|
|Animal models of autism: Pathogenesis and treatment||Powell, Craig||University of Texas Southwestern Medical Center|
|Novel genetic animal models of autism||Powell, Craig||University of Texas Southwestern Medical Center|
|Neuroligin function in vivo: Implications for autism and mental retardation||Powell, Craig||University of Texas Southwestern Medical Center|
|Genetic models of serotonin transporter regulation linked to mental disorders||Ramamoorthy, Sammanda||Medical University of South Carolina|
|Role of a novel Wnt pathway in autism spectrum disorders||Reichardt, Louis||University of California, San Francisco|
|A novel cell-based assay for autism research and drug discovery||Restifo, Linda||University of Arizona|
|Caspr2 dysfunction in autism spectrum disorders||Robbins, Elissa||Yale University|
|Novel strategies to manipulate Ube3a expression for the treatment of autism and Angelman syndrome||Roth, Bryan||University of North Carolina at Chapel Hill|
|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|
|Dissecting the neural control of social attachment||Shah, Nirao||University of California, San Francisco|
|Mice lacking Shank postsynaptic scaffolds as an animal model of autism||Sheng, Morgan||Massachusetts Institute of Technology|
|Using zebrafish and chemical screening to define function of autism genes||Sive, Hazel||Whitehead Institute for Biomedical Research|
|Characterization of autism susceptibility genes on chromosome 15q11-13||Smith, Stephen||Beth Israel Deaconess Medical Center|
|Identifying impairments in synaptic connectivity in mouse models of ASD||Speed, Haley||University of Texas Southwestern Medical Center|
|Function and dysfunction of neuroligins||Sudhof, Thomas||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.