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
Methods for production of ICSI and SCNT derived macaque 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
Animal models of autism: Pathogenesis and treatment 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
Novel genetic animal models of autism 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
Preclinical testing of novel oxytocin receptor activators in models of autism phenotypes Moy, Sheryl 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
Novel models to define the genetic basis of autism Mills, Alea Cold Spring Harbor Laboratory
16p11.2: defining the gene(s) responsible 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

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.