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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

Development of mGluR5 antagonists to treat fragile X syndrome and autism

Principal Investigator

Principal Investigator

Carpenter, Randall

Description

Description

Genetically engineered animal models have facilitated understanding of the effects of the mutations in the FMR1 gene that causes fragile X syndrome (FXS) on brain development and function. Studies with these animal models suggest that the symptoms of FXS reflect excessive protein synthesis downstream of mGluR5, a metabotropic glutamate receptor. Blocking this receptor with an antagonist could yield an effective drug treatment. This project will develop an existing lead compound, an mGluR5 antagonist, and advance it through the preclinical studies necessary to fulfill FDA requirements to open an Investigational New Drug application and perform initial testing in humans. These studies will test the hypothesis that mGluR5 antagonists can be effective treatments of FXS and other disorders of brain development, including autism.

Funder

Funder

National Institutes of Health

Fiscal Year Funding

Fiscal Year Funding

1048100

Current Award Period

Current Award Period

na

Strategic Plan Question

Strategic Plan Question

Question 4: Which Treatments And Interventions Will Help?

Strategic Plan Objective

Strategic Plan Objective

4O. Not specific to Question 4 objectives

Project Link

Project Link

Development of mGluR5 antagonists to treat fragile X syndrome and autism (External web link)

Institution

Institution

Seaside Therapeutics, LLC

State/Country

State/Country

Massachusetts

Project Number

Project Number

5U01MH078270-03

Federal or Private?

Federal or Private?

Federal

Received ARRA Funding?

Received ARRA Funding?

No

History/Related Projects

History/Related Projects

Development of MGLUR5 antagonists to treat fragile X syndrome and autism | 1068100 | 2008 | U01MH078270-02

 
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