Technology Listings

Compounds for the Treatment of Fragile X Syndrome and Methods of Screening


Small molecule compounds that may have therapeutic efficacy in Fragile X Syndrome and chemical genetic screens for potential drug targets.

Technical Summary

Fragile X Syndrome (FXS), the most common heritable form of mental retardation, is caused by transcriptional misregulation of the FMR1 gene and a subsequent loss of function of the RNA binding protein, fragile X mental retardation protein (FMRP). FXS is also the leading known cause of autism.

Currently there is no effective drug therapy for FXS, and there have been no FMR1-based assays amenable to candidate drug screens. Dr. Stephen Warren and his colleagues discovered glutamate toxicity in a Drosophila model of FXS and from their discovery, they developed the first chemical genetic screen for FXS. Using the screen, the researchers identified nine small molecule compounds that significantly rescued multiple phenotypes in FMR1 mutant flies.

Patent Information
App Type Country Serial No. Patent No. File Date Issued Date Expire Date
Divisional United States 14/641,832 3/9/2015    
Tech ID: 06011
Published: 2/24/2009

Rajsekhar Guddneppanavar
Licensing Associate
Emory University

Stephen Warren
Shuang Chang
Peng Jin

Genetic Disorders
Rare/Orphan Diseases
Repurposed Drug
Small Molecule