Emory University Archives

FMRP Regulatory Role in Human Hippocampal Development and Therapeutic Interventions in Fragile X

October 16, 2025

Fragile X syndrome hippocampal organoids show neuron–glia imbalance. This team will map disrupted gene networks and test PDE inhibitors to restore brain function.

Characterization and Modulation of microRNAs in Fragile X Syndrome

June 7, 2022

MicroRNAs are disrupted in Fragile X; the team will work to understand this and explore ways to correct it with drugs which directly target microRNAs.

Three-Dimensional Model for Identifying Fragile X Treatments

May 16, 2018

With a $90K FRAXA grant, Emory scientists are creating Fragile X brain organoids—3D human cell models—to reveal disease mechanisms and guide new treatments.

Functional Interplay Between FMRP and CDK5 Signaling

September 13, 2014

FRAXA-funded work showed CDK5 signaling is disrupted in Fragile X. CDK5 drugs are in development for Alzheimer’s so this pathway offers a promising new FX treatment angle.

Genetic and Pharmacologic Manipulation of PI3K Activity in FXS: Assessing Potential Therapeutic Value

September 2, 2012

Targeting the PI3K/mTOR cascade — specifically p110β — in Fragile X mice reversed neural and behavioral dysfunctions, validating it as a treatment pathway.

Small Molecule Screen Using Fragile X Neural Stem Cells

February 1, 2010

Researchers found that FMRP-deficient neural stem cells divide too much and fail to mature properly; screening compounds revealed candidates restoring normal behavior.