Jeannie Lee, MD, PhD and Chloe Chen, PhD

Modeling R-Loop Therapy for Fragile X Syndrome in Patient-Derived Brain Organoids

July 18, 2025

Fragile X syndrome researchers model R-loop therapy in patient-derived brain organoids to restore FMR1, accelerating a curative approach supported by FRAXA.

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Reactivating the FMR1 Gene to Reverse Fragile X Syndrome

November 7, 2023

This project aims to reactivate the FMR1 gene to combat Fragile X Syndrome, with the goal of restoring vital protein function. This work is now funded by a new FRAXA grant.

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Innovative Breakthrough in Fragile X Treatment: The Promise of Antisense Oligonucleotide (ASO) Therapy

June 27, 2023

This changes everything! FRAXA funded research introduces Antisense Oligonucleotide (ASO) Therapy, redefining Fragile X syndrome treatment and understanding.

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Andre Hoogeveen , PhD

Reactivating the FMR1 Gene

February 27, 2004

With $171.6K from FRAXA, Dr. Andre Hoogeveen’s Erasmus team developed methods to reactivate the Fragile X gene, advancing curative research.

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Dr. Eric Kandel, MD, Columbia University, FRAXA research grant

Transgenic Mouse Model Studies of Fragile X Syndrome

February 27, 2001

With $410K from FRAXA, Dr. Eric Kandel’s team at Columbia developed Fragile X mouse models to advance future studies of the disorder.

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FRAXA Funded Research

Current Research Grants (38)