mRNA Therapy for Fragile X Syndrome

Dr. Kathryn Whitehead, Associate Professor at Carnegie Mellon University, helped develop the revolutionary science behind the COVID-19 vaccines.

With a 2 year $100,000 grant from FRAXA, her team will now adapt mRNA technology to deliver the missing Fragile X protein, to treat people who have Fragile X syndrome.  

The COVID-19 pandemic has fueled the use of messenger RNA (mRNA) as a new class of medicine which has the potential to treat many disorders, including rare genetic disorders like Fragile X syndrome. Recent advances in mRNA technology, including modifications to the mRNA itself along with improvements to the delivery vehicle, have transformed the utility of mRNA as a potential therapy to restore or replace different types of therapeutic proteins.

Learn more about this research grant: https://www.fraxa.org/mrna-therapy-fo...

FRAXA’s mission is to find effective treatments and ultimately a cure for Fragile X syndrome. We directly fund research grants and fellowships at top universities around the world. We partner with biomedical and pharmaceutical companies, large and small, to bridge the gap between research discoveries and actual treatments.

Treatments for Fragile X are likely to help people affected by autism, Alzheimer’s, and other brain disorders.

To learn more about FRAXA's Fragile X syndrome research, to get involved with Fragile X awareness and events or to donate to FRAXA research, visit www.fraxa.org

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