Lee, Jeannie Archives

Fragile X Research Update: A Turning Point for Treatments and Curative Approaches

September 17, 2025

Fragile X research is at a turning point. FRAXA is funding ASO therapy and CRISPR-based gene reactivation to target the root cause of Fragile X.

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.

Dr. Jeannie Lee standing in front of a staircase at Harvard Medical School, recipient of the Blavatnik $1M award for Fragile X gene reactivation research

Harvard’s Dr. Jeannie Lee Wins $1M Award to Develop Gene Reactivation Therapy for Fragile X

May 30, 2025

Dr. Jeannie Lee wins $1M Blavatnik Award to advance FMR1 gene reactivation therapy for Fragile X, building on years of FRAXA-funded research.

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.

Unraveling Fragile X Syndrome: New Insights into FMR1 Gene Reactivation

June 20, 2023

Discover groundbreaking methods for reactivating the FMR1 gene in Fragile X syndrome. Dive into the transformational research and the implications of self-healing at a cellular level.

Can CRISPR Cure Fragile X Syndrome?

February 28, 2018

CRISPR/Cas9 was used by MIT researchers to remove the molecular tags that keep the mutant gene shut off in Fragile X syndrome neurons and resulted in some of them producing protein normally. Much work is being done right now, with exciting new discoveries coming at a fast and furious pace.

18th International Fragile X and Related Neurodevelopmental Disorders Workshop, Quebec, Canada

In Their Own Words: Reports From the International Fragile X Workshop

October 29, 2017

The 18th International Fragile X Workshop in Quebec was a great success, featuring more Fragile X research than ever before!

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.

$75,000 Raised for Fragile X Research by Friends of FRAXA at our Fall Fling

September 15, 2017

Over 150 friends joined FRAXA’s Fall Fling at Smith Barn, raising $75K for Fragile X research, including a $25K check hand-delivered!

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.

Fragile X Research Update: A Turning Point for Treatments and Curative Approaches

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

Harvard’s Dr. Jeannie Lee Wins $1M Award to Develop Gene Reactivation Therapy for Fragile X

Reactivating the FMR1 Gene to Reverse Fragile X Syndrome

Unraveling Fragile X Syndrome: New Insights into FMR1 Gene Reactivation

Can CRISPR Cure Fragile X Syndrome?

In Their Own Words: Reports From the International Fragile X Workshop

Investigating Gene Reactivation to Treat Fragile X Syndrome

$75,000 Raised for Fragile X Research by Friends of FRAXA at our Fall Fling

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

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