Strategic Plan Objective Detail
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Question 4: Short-term Objective B  

$23,229,501.04
Fiscal Year: 2010

Green dot: Objective has greater than or equal to the recommended funding.4SB. 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.

Download 2010 Question 4: Short-term Objective B projects (EXCEL)
Note: Initial Sort is by Principal Investigator. Sorting by other columns is available by clicking on the desired column header.
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

Objective Cumulative Funding Table

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
4.5
$15,879,827
42 projects

4.S.B
$20,162,709
70 projects

4.S.B
$23,229,501
92 projects

4.S.B
$21,606,118
89 projects

4.S.B
$21,232,514
94 projects

$102,110,669
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.