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.

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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.

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Towards Understanding the Role of FMRP in Human Brain Development Using Brain Organoids

December 8, 2020

Dr. Zhexing Wen and Dr. Peng Jin of the newly funded Fragile X Center of Excellence at Emory University School of Medicine join us in this seminar to present about Understanding the Role of FMRP in Human Brain Development Using Brain Organoids.

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Todd-Haenfler

CRISPR Reactivation of the Fragile X Gene

December 6, 2017

“We are trying to target the first event that goes wrong in Fragile X syndrome”, says Todd, “One reason our previous attempts to develop treatments for Fragile X syndrome have failed is that they’ve tried to target the downstream effects of losing the Fragile X protein. The protein does many things… bypassing all the functions that it normally takes care of has proven difficult from a pharmacologic perspective.”

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Jeannie Lee - Fragile X researcher

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.

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Genome-wide Epigenetic Markers in Fragile X

February 5, 2009

With $45,000 in grants from FRAXA Research Foundation over several years, Dr. Miklos Toth of Cornell University studied epigenetics (ie factors other than the gene itself) which can determine symptom severity in Fragile X.

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FRAXA Funded Research

Current Research Grants (37)