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

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)
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Project Title Principal Investigator Institution
Neuropharmacology of motivation and reinforcement in mouse models of autistic spectrum disorders Malanga, C.J. University of North Carolina School of Medicine
Evaluation of altered fatty acid metabolism via gas chromatography/mass spectroscopy and time-of-flight secondary ion mass spectroscopy imaging in the propionic acid rat model of autism spectrum disorders MacFabe, Derrick University of Western Ontario
Autism iPSCs for studying function and dysfunction in human neural development Loring, Jeanne The Scripps Research Institute
Insight into MeCP2 function raises therapeutic possibilities for Rett syndrome Lomvardas, Stavros University of California, San Francisco
Using Drosophila to model the synaptic function of the autism-linked NHE9 Littleton, J. Troy Massachusetts Institute of Technology
Interaction between MEF2 and MECP2 in the pathogenesis of autism spectrum disorders - 1 Lipton, Stuart Burnham Institute
Serotonin, corpus callosum, and autism Lin, Rick University of Mississippi Medical Center
The genetics of restricted, repetitive behavior: An inbred mouse model Lewis, Mark University of Florida
Behavioral and physiological consequences of disrupted Met signaling Levitt, Pat University of Southern California
Shank3 mutant characterization in vivo Kouser, Mehreen University of Texas Southwestern Medical Center
Animal model of speech sound processing in autism Kilgard, Michael University of Texas at Dallas
Control of synaptic protein synthesis in the pathogenesis and therapy of autism Kelleher, Raymond Massachusetts General Hospital
Investigating the effects of chromosome 22q11.2 deletions Karayiorgou, Maria Columbia University
Functional study of synaptic scaffold protein SHANK3 and autism mouse model Jiang, Yong-Hui Duke University
Preclinical testing of novel oxytocin receptor activators in models of autism phenotypes Jarstfer, Michael University of North Carolina at Chapel Hill
Neurogenomics in a model for procedural learning Hilliard, Austin University of California, Los Angeles
High content screens of neuronal development for autism research Halpain, Shelley University of California, San Diego
Exploring the neuronal phenotype of autism spectrum disorders using induced pluripotent stem cells Hallmayer, Joachim; Dolmetsch, Ricardo Stanford University
Genomic imbalances at the 22q11 locus and predisposition to autism Gogos, Joseph Columbia University
Identification of autism genes that regulate synaptic Nrx/Nlg signaling complexes Garner, Craig Stanford University
Deriving neuroprogenitor cells from peripheral blood of individuals with autism Fujinami, Robert University of Utah
Functional genomic dissection of language-related disorders Fisher, Simon University of Oxford
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
Patient iPS cells with copy number variations to model neuropsychiatric disorders Ellis, James The Hospital for Sick Children

Objective Multiyear 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
42 projects

70 projects

92 projects

89 projects

94 projects

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.