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
Investigation of the role of MET kinase in autism Dawson, Ted Johns Hopkins University School of Medicine
Control of synaptic protein synthesis in the pathogenesis and therapy of autism Kelleher, Raymond Massachusetts General Hospital
Neurobiology of mouse models for human chr 16p11.2 microdeletion and fragile X Bear, Mark Massachusetts Institute of Technology
Development of a high-content neuronal assay to screen therapeutics for the treatment of cognitive dysfunction in autism spectrum disorders Bear, Mark Massachusetts Institute of Technology
Mice lacking Shank postsynaptic scaffolds as an animal model of autism Sheng, Morgan Massachusetts Institute of Technology
Neural and cognitive mechanisms of autism Sur, Mriganka Massachusetts Institute of Technology
Dissecting the circuitry basis of autistic-like behaviors in mice Feng, Guoping Massachusetts Institute of Technology
Synaptic and circuitry mechanisms of repetitive behaviors in autism Feng, Guoping Massachusetts Institute of Technology
Using Drosophila to model the synaptic function of the autism-linked NHE9 Littleton, J. Troy Massachusetts Institute of Technology
Genetic models of serotonin transporter regulation linked to mental disorders Ramamoorthy, Sammanda Medical University of South Carolina
The role of SHANK3 in autism spectrum disorders Buxbaum, Joseph Mount Sinai School of Medicine
A preclinical model for determining the role of AVPR1A in autism spectrum disorders Charles, Rhonda Mount Sinai School of Medicine
Animal models of neuropsychiatric disorders Crawley, Jacqueline National Institutes of Health
Regulation of gene expression in the brain Young, Walter National Institutes of Health
Synaptic plasticity, memory and social behavior Chevere-Torres, Itzamarie New York University
Methods for production of ICSI and SCNT derived macaque stem cells Schatten, Gerald Southwest Foundation For Biomedical Research
Optimization of methods for production of both ICSI- and SCNT derived baboon embryonic stem cells Schatten, Gerald Southwest Foundation For Biomedical Research
Micro-RNA regulation in pluripotent stem cells Schatten, Gerald Southwest Foundation 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
Exploring the neuronal phenotype of autism spectrum disorders using induced pluripotent stem cells Hallmayer, Joachim; Dolmetsch, Ricardo Stanford University
Synaptic deficits of iPS cell-derived neurons from patients with autism Mao, Rong Stanford University
Function and dysfunction of neuroligins Sudhof, Thomas Stanford University
Identification of autism genes that regulate synaptic Nrx/Nlg signaling complexes Garner, Craig Stanford University
Role of L-type calcium channels in hippocampal neuronal network activity Owen, Scott Stanford 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.