Project Detail
Interagency Autism Coordinating Committee (IACC) logo
Office of Autism Research Coordination (OARC) logo

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

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. Project Status


Funder Autism Speaks
Fiscal Year Funding $60,000.00
Current Award Period 2009-2011
Project Number 3769
Principal Investigator Restifo, Linda
Received ARRA Funding? No
Strategic Plan Question Question 4: Which Treatments And Interventions Will Help? (Treatments)
Subcategory Model Systems/Therapeutic Targets
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.
Federal or Private? Private
Institution University of Arizona
State/Country Arizona
Web Link 1 A novel cell-based assay for autism research and drug discovery (External web link)
Web Link 2 No URL available.
Web Link 3 No URL available.
New! History/Related Projects A novel cell-based assay for autism research and drug discovery | $60,000.00 | 2009 | 3769
A novel cell-based assay for autism research and drug discovery | $60,000.00 | 2008 | 3769