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
Optimization of methods for production of both ICSI- and SCNT derived baboon embryonic stem cells Schatten, Gerald Southwest Foundation For Biomedical Research
NrCAM, a candidate susceptibility gene for visual processing deficits in autism Maness, Patricia 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
Novel probiotic therapies for autism Patterson, Paul California Institute of Technology
Novel models to define the genetic basis of autism Mills, Alea Cold Spring Harbor Laboratory
Novel genetic animal models of autism Powell, Craig University of Texas Southwestern Medical Center
Neuropharmacology of motivation and reinforcement in mouse models of autistic spectrum disorders Malanga, C.J. University of North Carolina School of Medicine
Neuroligin function in vivo: Implications for autism and mental retardation Powell, Craig University of Texas Southwestern Medical Center
Neurogenomics in a model for procedural learning Hilliard, Austin University of California, Los Angeles
Neurogenetic model of social behavior heterogeneity in autism spectrum disorders Platt, Michael Duke University
Neurobiology of sociability in a mouse model system relevant to autism Brodkin, Edward University of Pennsylvania
Neurobiology of mouse models for human chr 16p11.2 microdeletion and fragile X Bear, Mark Massachusetts Institute of Technology
Neurobiological mechanism of 15q11-13 duplication autism spectrum disorder Anderson, Matthew Beth Israel Deaconess Medical Center
Neural mechanisms of social cognition and bonding Young, Larry Emory University
Neural mechanisms of social cognition and bonding Young, Larry Emory University
Neural and cognitive mechanisms of autism Sur, Mriganka Massachusetts Institute of Technology
Murine genetic models of autism Veenstra-Vanderweele, Jeremy Vanderbilt University
Mouse genetic model of a dysregulated serotonin transporter variant associated with autism Veenstra-Vanderweele, Jeremy Vanderbilt University
Modeling and pharmacologic treatment of autism spectrum disorders in Drosophila McDonald, Thomas Albert Einstein College of Medicine of Yeshiva University
Micro-RNA regulation in pluripotent stem cells Schatten, Gerald Southwest Foundation For Biomedical Research
Mice lacking Shank postsynaptic scaffolds as an animal model of autism Sheng, Morgan Massachusetts Institute of Technology
Methods for production of ICSI and SCNT derived macaque stem cells Schatten, Gerald Southwest Foundation For Biomedical Research
Investigation of the role of MET kinase in autism Dawson, Ted Johns Hopkins University School of Medicine
Investigating the effects of chromosome 22q11.2 deletions Karayiorgou, Maria Columbia University

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.