Stylized blue brain with an orange highlighted region representing the nucleus accumbens in Fragile X syndrome research

Bridging Naturalistic Behavior and Nucleus Accumbens in Fragile X Syndrome

How do sex and gene dosage interact to shape brain function and behavior in Fragile X syndrome? The team aims to uncover why symptoms differ between males and females.

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Eric Wang and Alana O’Brien, researchers at the University of Florida working on an FMRP autoregulation gene therapy project for Fragile X syndrome.

Harnessing FMRP Autoregulation for Safer Gene Therapy in Fragile X

Fragile X syndrome gene therapy could be safer with an FMRP “safety dial” that self-regulates expression using natural FMR1 control elements.

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Fragile X Unplugged: Establishing Mobile EEG as the Next Frontier

A $100,000 FRAXA grant to Cincinnati Children’s Hospital is simplifying and testing EEG technology for home use, improving clinical trial accessibility and efficiency.

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FRAXA-DVI research team in the lab conducting preclinical Fragile X drug validation studies

FRAXA Drug Validation Initiative (FRAXA-DVI)

The FRAXA Drug Validation Initiative (FRAXA-DVI) provides streamlined, cost-effective preclinical testing to evaluate investigational and repurposed compounds for Fragile X syndrome.

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Drs. Oswald Steward and Patricia Pirbhoy in their laboratory, studying RNA localization and gene activity in Fragile X syndrome using spatial transcriptomics.

Uncovering the Dynamics of FMR1 Using Spatial Transcriptomics for Fragile X

Scientists investigate how changes in RNA localization affect cognition and behavior in Fragile X, building knowledge toward more precise therapies.

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Investigating the Role of the Insulin Degrading Enzyme (IDE) in Fragile X Syndrome

This study explores how disrupted insulin signaling affects metabolism and brain function in Fragile X, revealing new treatment targets for both body and mind.

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Mark Bear, PhD & Sara Kornfeld Simpson

Altered Physiology of Primary Visual Cortex in Fragile X Syndrome

This team believes inhibitory neurons expressing somatostatin are impaired in Fragile X. They will see if stimulating these neurons has therapeutic potential.

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The Endocannabinoid System and Fragile X Syndrome

This project will examine how CBD and other drugs targeting the endocannabinoid system affect hyperexcitable Fragile X neurons to identify new treatment strategies.

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FRAXA Funded Research

Current Research Grants (38)