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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FRAXA Drug Validation Initiative (FRAXA-DVI)

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

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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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FRAXA Funded Research

Current Research Grants (38)