Fragile X deficits in mice respond to gene therapy

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A gene therapy developed at the University of California, Riverside, successfully restored normal brain activity and behavior in a mouse model of fragile X syndrome by replacing a missing brain protein. The study suggests potential for gene therapy to address the root causes of this condition.

A gene therapy designed to replace a missing brain protein restored normal brain activity and improved behavior in a mouse model of fragile X syndrome (FXS), according to a study led by researchers at the University of California, Riverside. The findings, published in Molecular Therapy Nucleic Acids, suggest that gene therapy may one day address the underlying cause of FXS rather than simply treating its symptoms.