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
Patient iPS cells with copy number variations to model neuropsychiatric disorders Ellis, James The Hospital for Sick Children
Serotonin, autism, and investigating cell types for CNS disorders Dougherty, Joseph The Rockefeller University
Autism iPSCs for studying function and dysfunction in human neural development Loring, Jeanne The Scripps Research Institute
Small-molecule compounds for treating autism spectrum disorders Philpot, Ben The University of North Carolina at Chapel Hill
A novel cell-based assay for autism research and drug discovery Restifo, Linda University of Arizona
Basal ganglia circuitry and molecules in pathogenesis of motor stereotypy Yang, Xiangdong University of California, Los Angeles
Neurogenomics in a model for procedural learning Hilliard, Austin University of California, Los Angeles
Cntnap2 in a behavioral model of autism White, Stephanie University of California, Los Angeles
High content screens of neuronal development for autism research Halpain, Shelley University of California, San Diego
Insight into MeCP2 function raises therapeutic possibilities for Rett syndrome Lomvardas, Stavros University of California, San Francisco
Dissecting the neural control of social attachment Shah, Nirao University of California, San Francisco
Role of a novel Wnt pathway in autism spectrum disorders Reichardt, Louis University of California, San Francisco
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
A mouse knock-in model for ENGRAILED 2 autism susceptibility Millonig, James University of Medicine & Dentistry of New Jersey
Serotonin, corpus callosum, and autism Lin, Rick University of Mississippi Medical Center
NrCAM, a candidate susceptibility gene for visual processing deficits in autism Maness, Patricia 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 Pedersen, Cort 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
Novel strategies to manipulate Ube3a expression for the treatment of autism and Angelman syndrome Roth, Bryan University of North Carolina at Chapel Hill
Characterization of a novel mouse model of restricted repetitive behaviors Moy, Sheryl University of North Carolina at Chapel Hill
Neuropharmacology of motivation and reinforcement in mouse models of autistic spectrum disorders Malanga, C.J. University of North Carolina School of Medicine
Functional genomic dissection of language-related disorders Fisher, Simon University of Oxford
Neurobiology of sociability in a mouse model system relevant to autism Brodkin, Edward University of Pennsylvania

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.