|Project Title||Principal Investigator||Institution|
|Micro-RNA regulation in pluripotent stem cells||Schatten, Gerald||Southwest Foundation For Biomedical Research|
|Novel strategies to manipulate Ube3a expression for the treatment of autism and Angelman syndrome||Roth, Bryan||University of North Carolina at Chapel Hill|
|Caspr2 dysfunction in autism spectrum disorders||Robbins, Elissa||Yale University|
|A novel cell-based assay for autism research and drug discovery||Restifo, Linda||University of Arizona|
|Role of a novel Wnt pathway in autism spectrum disorders||Reichardt, Louis||University of California, San Francisco|
|Genetic models of serotonin transporter regulation linked to mental disorders||Ramamoorthy, Sammanda||Medical University of South Carolina|
|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|
|Animal models of autism: Pathogenesis and treatment||Powell, Craig||University of Texas Southwestern Medical Center|
|High-resolution diffusion tensor imaging in mouse models relevant to autism||Poptani, Harish||University of Pennsylvania|
|Neurogenetic model of social behavior heterogeneity in autism spectrum disorders||Platt, Michael||Duke University|
|Small-molecule compounds for treating autism spectrum disorders||Philpot, Ben||The University of North Carolina at Chapel Hill|
|Preclinical testing of novel oxytocin receptor activators in models of autism phenotypes||Pedersen, Cort||University of North Carolina at Chapel Hill|
|Novel probiotic therapies for autism||Patterson, Paul||California Institute of Technology|
|Role of L-type calcium channels in hippocampal neuronal network activity||Owen, Scott||Stanford University|
|Systematic analysis of neural circuitry in mouse models of autism||Osten, Pavel||Cold Spring Harbor Laboratory|
|Interaction between MEF2 and MECP2 in the pathogenesis of autism spectrum disorders -2||Nakanishi, Nobuki||Burnham Institute|
|Characterization of a novel mouse model of restricted repetitive behaviors||Moy, Sheryl||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|
|16p11.2: defining the gene(s) responsible||Mills, Alea||Cold Spring Harbor Laboratory|
|Novel models to define the genetic basis of autism||Mills, Alea||Cold Spring Harbor Laboratory|
|A mouse knock-in model for ENGRAILED 2 autism susceptibility||Millonig, James||University of Medicine & Dentistry of New Jersey|
|Modeling and pharmacologic treatment of autism spectrum disorders in Drosophila||McDonald, Thomas||Albert Einstein College of Medicine of Yeshiva University|
|Synaptic deficits of iPS cell-derived neurons from patients with autism||Mao, Rong||Stanford University|
|NrCAM, a candidate susceptibility gene for visual processing deficits in autism||Maness, Patricia||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.