Gene Therapy Restores Brain Function in Fragile X Syndrome Mouse Model

Researchers at the University of California, Riverside have successfully used gene therapy to replace a missing brain protein in mice with fragile X syndrome (FXS), restoring normal brain activity and improving behavioral symptoms. The study, published in Molecular Therapy Nucleic Acids, demonstrates that this therapeutic approach targets the underlying genetic cause of FXS rather than merely managing its symptoms. Fragile X syndrome is the most common inherited cause of intellectual disability and autism spectrum disorder, affecting thousands of individuals worldwide. This breakthrough suggests that gene therapy could potentially offer a disease-modifying treatment for FXS patients in the future, moving beyond current symptomatic management strategies.