mRNA Therapy for Fragile X Syndrome
Principal Investigator
FRAXA Fellow
Pittsburgh, PA
Summary
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 Science
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.
Lack of a single protein, FMRP, causes Fragile X syndrome and is linked with other autism spectrum disorders. With this FRAXA research grant, Dr. Saigopalakrishna Yerneni and Professor Kathryn Whitehead will take the first steps in creating a gene therapy solution for the treatment of Fragile X syndrome. They will create this treatment by creating a drug delivery system based on lipid nanoparticles that deliver mRNA encoding FMRP to brain cells. Successful expression of this protein is anticipated to alleviate disease symptoms.
The team anticipates that resulting data will set the stage for follow-on funding for in vivo studies in mice and eventually larger animal models and open a new avenue of target-specific mRNA therapy development for brain disorders such as FXS.
Meet the Scientist
In a 2021 TED Talk, Dr. Kathryn Whitehead answers the question: what if you were holding life-saving medicine, but had no way to administer it? Her work helped build COVID-19 vaccines and is forging a path for future mRNA-based therapies.
