Harvard University Archives

Jeannie Lee, MD, PhD and Chloe Chen, PhD

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

June 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.

Reactivating the FMR1 Gene to Reverse Fragile X Syndrome

November 7, 2023

FRAXA Research Foundation is dedicated to funding breakthrough research, providing $240,000 to reactivate the FMR1 gene to combat Fragile X Syndrome, with the goal of restoring vital protein function and advancing towards a cure.

Investigating Gene Reactivation to Treat Fragile X Syndrome

September 22, 2017

With a $180,000 grant from FRAXA Research Foundation from 2016-2017, Dr. Jeannie Lee and her team at Harvard are working to reactivate the gene that is silenced in Fragile X syndrome.

Neural Markers of Fragile X: A Powerful New Tool for Clinical Trials

August 17, 2017

Once the neural marker is identified for a particular challenge, such as kids with poor language versus good language, neural markers can be measured during drug and behavioral therapy trials to see if a child is improving based on objective biological measures.

The X Factor – Turning on X Chromosome Genes to Treat X-linked Disorders

May 9, 2016

Harvard researcher Jeannie T. Lee, MD, PhD, moves closer to turning on select genes on the X chromosome to treat people with X-linked disorders.

Stephen Haggarty, PhD, Harvard/MIT, Principal Investigator, FRAXA research grant

Small Molecule Modulators of Lithium for Treatment of Fragile X Syndrome

February 1, 2010

With a $219,500 grant from FRAXA Research Foundation, Dr. Stephen Haggarty from Havard/MIT developed a high-throughput drug screen to find compounds that inhibit GSK3, a critical enzyme in Fragile X. He looked for compounds that can accomplish this either alone or in combination with lithium, offering the possibility of enhancing the effectiveness of lithium as a treatment. His drug screen used patient-specific neural progenitor (NP) cells derived from human induced pluripotent stem cells (iPSCs) – which are created from cells in a skin biopsy from people with Fragile X syndrome (FXS) and other autism spectrum disorders.

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

Reactivating the FMR1 Gene to Reverse Fragile X Syndrome

Investigating Gene Reactivation to Treat Fragile X Syndrome

Neural Markers of Fragile X: A Powerful New Tool for Clinical Trials

The X Factor – Turning on X Chromosome Genes to Treat X-linked Disorders

Small Molecule Modulators of Lithium for Treatment of Fragile X Syndrome

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