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Interagency Autism Coordinating Committee (IACC)
Autism Research Database
Office of Autism Research Coordination (OARC)
 
Project Element Element Description

Project Title

Project Title

A novel cell-based assay for autism research and drug discovery

Principal Investigator

Principal Investigator

Restifo, Linda

Description

Description

In the present study, researchers aim to develop a cell-based system for identifying how neurons are affected by mutations in autism-related genes. This cell-based system will be used in a screen to identify drugs able to reverse any observed changes caused by these mutations. Dr. Restifo and colleagues will focus on the effects of three genes that have been found to be associated with autism in humans. They will create genetically modified fruit flies (an animal often used to study basic neuronal development) which have mutations in the fly genes that correspond to the human genes which are mutated in autism. Neurons from these flies will be analyzed for any abnormalities in size and shape that result from mutating these genes. Then, a selection of Food and Drug Administration-approved drugs will be tested to determine whether any can restore normal growth patterns to the mutant neurons. This research, if successful, will advance knowledge of the effects of autism-associated mutations on neuronal development and could create a novel system for the identification of drugs which might eventually be used to treat human patients with autism.

Funder

Funder

Autism Speaks

Fiscal Year Funding

Fiscal Year Funding

0

Current Award Period

Current Award Period

2008-2012

Strategic Plan Question

Strategic Plan Question

Question 4: Which Treatments And Interventions Will Help?

Strategic Plan Objective

Strategic Plan Objective

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.

Project Link

Project Link

A novel cell-based assay for autism research and drug discovery (External web link)

Institution

Institution

University of Arizona

State/Country

State/Country

Arizona

Project Number

Project Number

3769

Federal or Private?

Federal or Private?

Private

Received ARRA Funding?

Received ARRA Funding?

No

History/Related Projects

History/Related Projects

A novel cell-based assay for autism research and drug discovery | 60000 | 2009 | 3769
A novel cell-based assay for autism research and drug discovery | 60000 | 2010 | 3769
A novel cell-based assay for autism research and drug discovery | 60000 | 2008 | 3769

 
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