Curative Therapies for Fragile X: Advancing Gene and Protein Treatments
Curative therapies are one of FRAXA’s highest priorities, representing a focused effort to move beyond symptom management and toward lasting change for individuals with Fragile X syndrome.
Gene therapy, gene editing, mRNA delivery, protein replacement, and antisense oligonucleotides (ASOs) are leading the way. These approaches address the underlying cause of Fragile X. They are bold, innovative, and focused on long-term solutions.
This kind of work doesn’t start from zero. Years of FRAXA-funded research have built the foundation. Now, with better tools and deeper understanding, today’s projects are moving faster and with more precision, bringing curative strategies closer to reality.
Funding Opportunities for Researchers
The Curative Therapies Research Program offers flexible grants of up to $100,000 per year, with applications accepted on a rolling basis. There are no rigid structures. Projects can support principal investigators, trainees, supplies, or specialized tools, as long as the focus is clear: developing effective therapies for Fragile X.
Visit the FRAXA Grant Application page to learn more and get started.
Changing the Future of Fragile X
Curative therapies are already reshaping the research landscape in Fragile X syndrome.
By advancing this work, we are helping build a future where effective, durable treatments are within reach.
You can help accelerate this progress. Every donation brings these therapies closer to reality, turning scientific promise into real hope for families living with Fragile X.
Gratitude for the Families Driving Curative Research Forward
We are deeply grateful to the Dailey, Gale, Schatz, Berg, Pierce and Schutz families, whose extraordinary efforts have helped push FRAXA’s curative therapies program forward. These families have not only shared their journeys with Fragile X but have also raised significant funds to accelerate research with the greatest potential for lasting impact.
Collectively, these families have raised over $400,000 to help bring new treatments for Fragile X closer to reality.
Their generosity and determination inspire everything we do. Together, we are moving toward a future where curative treatments — and ultimately a cure — are within reach.
Help Turn Hope Into a Cure for Fragile X
“We’re on the brink of huge breakthroughs. Every donation to FRAXA fuels research that brings us closer to answers for families like ours and offers hope for a future free from Fragile X.”
Erich and Lexie Schutz
Advancing Curative Therapies
Below, you’ll find the most recent FRAXA-funded research exploring innovative strategies to restore or replace FMRP. From gene reactivation to mRNA and ASO therapies, these projects represent real momentum and real hope.
Modeling R-Loop Therapy for Fragile X Syndrome in Patient-Derived Brain Organoids
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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Gene Editing of FMR1 to Correct FXS Phenotypes in Mice
FRAXA awarded a $100,000 grant to Dr. Davidson and Dr. Yrigollen at Children’s Hospital of Philadelphia to explore gene editing tools aimed at treating Fragile X syndrome by correcting FMR1 mutations in a unique mouse model.
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Reactivating the FMR1 Gene to Reverse Fragile X Syndrome
FRAXA Research Foundation is dedicated to funding breakthrough research, providing $240,000 to reactivate the FMR1 gene to combat Fragile X Syndrome, with the goal of restoring vital protein function and advancing towards a cure.
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Antisense Oligonucleotides (ASOs) to restore FMRP in Human Fragile X Cerebral Organoids
Explore Dr. Richter’s encouraging results with ASOs for Fragile X syndrome. A $100,000 grant now fuels pivotal studies needed to advance toward ASO therapy.
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A Tat-Conjugate Approach to Treat Fragile X Syndrome
Explore Turner Lab’s novel approach to developing definitive treatment for Fragile X syndrome with a Tat-conjugated, truncated FMRP protein. This innovative strategy aims to restore brain circuit function and reduce abnormal behaviors linked to Fragile X by directly addressing the cause of Fragile X: a missing protein.
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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 $103,000 grant from FRAXA, her team will now adapt this technology to deliver the missing Fragile X protein, to treat people who have Fragile X syndrome.
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Genome-wide Screen for FMR1 Reactivation in Human FXS Neural Cells
Drs. Mahmoud Pouladi and Kagistia Utami at the Agency for Science, Technology and Research (A*STAR) in Singapore were awarded a $67,500 research grant from FRAXA Research Foundation and that led to much greater governmental funding to expand this work. Their goal is to reactivate the gene which is silenced in people who have Fragile X syndrome.
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Gene Therapy Translational Studies for Fragile X Syndrome
With this $90,000 award from FRAXA Research Foundation, Drs. Ernest Pedapati, Christina Gross, and student Lindsay Beasley will pursue preclinical gene therapy approaches using AAV (adeno-associated virus) vectors for treating Fragile X syndrome at Cincinnati Children’s Hospital. Dr. Craig Erickson elaborates about this in this video.
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Reintroducing FMRP via Tat to Reduce Symptoms of Fragile X Syndrome
FRAXA Research Foundation and the Fragile X Research Foundation of Canada awarded a grant of $100,000 over two years to Dr. Raymond Turner at the University of Calgary in Alberta, Canada. Dr. Turner and postdoctoral fellow Xiaoqin Zhan, PhD are attempting to reactivate a segment of FMRP to reverse symptoms of Fragile X in a mouse model of the disease to reduce abnormal behaviors.
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