The FRAXA Ingenuity Award recognizes someone taking an innovative approach to tackling Fragile X, despite daunting challenges and long odds.
This year’s Ingenuity Award goes to Dr. Kathryn Whitehead who is an Associate Professor in the Department of Chemical Engineering at Carnegie Mellon University. She helped develop the revolutionary science behind the COVID-19 vaccines and is now forging a path for future mRNA-based therapies for rare diseases. With a grant from FRAXA, Dr. Whitehead and her team are taking the first steps toward a new treatment for Fragile X syndrome by adapting mRNA technology to deliver the missing Fragile X protein. They are creating a drug delivery system based on lipid nanoparticles to deliver mRNA encoding FMRP to brain cells. Successful expression of this protein is anticipated to reverse Fragile X symptoms.
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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Негізгі бет Dr. Kathryn Whitehead - FRAXA’s Ingenuity Award recipient
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