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

January 24, 2014

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

September 2, 2013

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

February 22, 2005

A $70K FRAXA grant helped Drs. Thomas Tuschl and Neil Renwick study FMRP–miRNA links to identify better treatment targets for Fragile X.

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Fragile X Syndrome and RNAi

February 27, 2004

With $75K from FRAXA, Dr. Richard Carthew at Northwestern studied how RNA interference (RNAi) and FMRP interact in Fragile X.

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Fruit Fly Helps Reveal the Secrets of Fragile X

February 26, 2004

FRAXA’s $50K grant supported Dr. Bassem Hassan’s Flanders team in using fruit flies to model Fragile X and guide future therapy studies.

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

Understanding the Function of Fragile X Protein in Drosophila

February 26, 2003

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

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