Small Molecules To Target r(CGG) Expansions to Treat Fragile X Syndrome

FRAXA-funded scientists created small molecules that target the CGG repeat “off-switch” in Fragile X, aiming to restore the missing FMRP protein at its source.

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Kendal Broadie

Matrix Metalloproteinase Therapeutic Treatments for Fragile X Syndrome

Dr. Broadie showed that MMP enzymes disrupt synapse development in Fragile X. MMP inhibitors (e.g. minocycline) improved connectivity and behavior in fruit flies.

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Thomas Tuschl, PhD, at Rockefeller University, FRAXA research grant

Role of MicroRNAs in Fragile X Syndrome

With a $70,000 grant from FRAXA Research Foundation from 2004-2005, Drs. Thomas Tuschl and Neil Renwick and their team at Rockefeller University researched how FMRP interacts with miRNA in order to determine more effective treatment targets for Fragile X syndrome.

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Fragile X Syndrome and RNAi

With a $75,000 grant from FRAXA Research Foundation from 2003-2004, Dr. Richard Carthew and his team at Northwestern University studied their interest in gene expression by investigating the role of the recently discovered process of interfering RNA (RNAi). FMRP appears to be involved in the metabolism of RNAi, and may have a role in regulating the process; likewise, deficits in RNAi may contribute to the disease process in Fragile X.

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Fruit Fly Helps Reveal the Secrets of Fragile X

With a $50,000 grant from FRAXA Research Foundation from 2003-2004, Dr. Bassem Hassan and his team at Flanders University researched how Fragile X fruit flies can help further future studies for Fragile X treatment research.

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Haruhiko Siomi, PhD, at Tokushima University, FRAXA research grant

Understanding the Function of Fragile X Protein in Drosophila

FRAXA-funded Tokushima researchers mapped the Drosophila FMR1 gene and its pathways, revealing how Fragile X disrupts cell function.

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FRAXA Funded Research

Current Research Grants (37)