Dr. Lee’s team is testing novel gene editing therapies for Fragile X, aiming to repair FMR1 and restore the missing protein — a promising new strategy which is targeted and reversible.
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.
Explore the potential of ASOs in treating Fragile X syndrome & FXTAS. Dive into a comprehensive Q&A addressing key questions and breakthrough findings.
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.
Explore the latest breakthroughs in Fragile X research unveiled at the recent Banbury Meeting. Discover novel strategies, from gene therapy to protein replacement, that bring hope for curative therapies.
Fragile X syndrome might be treated by reactivating the gene which is shut down in the syndrome. Researchers were able to reduce FXS symptoms by inserting the FMR1 gene into the brains of young mice.
Each year, FRAXA funds a diverse portfolio of research. FRAXA Fellowships and Grants are seed funding for the future, the feedstock for the Fragile X treatment development pathway.
Dr. Xinyu Zhao of the Waisman Center and Department of Neuroscience at University of Wisconsin-Madison joins us in this seminar to present Interrogate the Functions of FMRP in Brain Development Using Stem Cells.
FRAXA Investigator Peter Todd, MD, PhD, tells us about CRISPR in Fragile X research, how realistic is it that it could turn the Fragile X gene back on, and if it can really cure Fragile X.
This FRAXA-funded team at Stanford University found that the Fragile X mutation impairs homeostatic plasticity in human neurons, by blocking synaptic retinoic acid signaling.
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.
Using CRISPR to reactivate FMRP, “We are trying to target the first event that goes wrong in Fragile X syndrome”, says FRAXA Investigator, Dr. Peter Todd.
FRAXA funded Dr. Peter Todd to use CRISPR to reactivate FMR1. Published results confirmed restored gene expression, a big step toward disease-modifying therapy.
With a $180,000 grant from FRAXA Research Foundation, Dr. Jeannie Lee and her team at Harvard are working to reactivate the gene that is silenced in Fragile X syndrome.
University of Michigan scientists Peter Todd, MD, PhD, and postdoctoral fellow Jill Haenfler, Ph.D., are adapting CRISPR to reactivate the FMR1 gene to reverse Fragile X syndrome.
4 Countries – 10 Teams – $1 Million for finding treatment targets, pinpointing outcome measures for clinical trials, and attempting to reactivate the gene which is silenced in Fragile X syndrome.
CRISPR offers the tantalizing possibility of “editing” genes precisely, and it could (theoretically) excise the methylated trinucleotide repeat sequence from Fragile X cells, returning them to normal.
