Cellular-Specific Therapeutic Targeting of Inhibitory Circuits in Fragile X Syndrome

Studies have shown that the function of inhibitory networks is disturbed in Fragile X. This abnormality is not well understood but appears to be secondary to abnormalities in metabotropic glutamate and endocannabinoid systems. With a $90,000 grant from FRAXA, Dr. Molly Huntsman’s team examined how these networks interact and how inhibitory deficits can best be remedied.

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Social Behavior as an Outcome Measure for Fragile X Clinical Trials

One of the features of the Fragile X mouse model which is relevant to the human Fragile X syndrome (and autism) is social behavior. Several tests show consistent social behavioral abnormalities in the Fragile X mouse model. With a $140,000 grant from FRAXA Research Foundation in 2012-2013, Dr. Willemsen at Erasmus University used social behavior tests to measure the effectiveness of several drug strategies.

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Improving Memory Reversal Testing and Treatment with AMPAkines in the Fragile X Knock-Out Mouse

W Ted Brown, MD, PhD

A two year $62,000 FRAXA grant was been awarded to W. Ted Brown, MD, PhD from 1997-1998. The Principal Investigator at the Institute For Basic Research in New York aims to develop an improved test to show learning deficits in the FMR1 knock-out mouse model of Fragile X, and test experimental drugs (Ampakines) that may be effective in treating these deficits.

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