A microglial basis for fragile X syndrome via impairment of FMRP-regulated lysosomal function

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18 Oct 2026 0 comments

Silencing of the FMRP-encoding FMR1 gene results in fragile X syndrome (FXS), the most common inheritable intellectual disability and autism. FMRP regulates various neuronal functions but clinical trials targeting neurons have failed, suggesting unrecognized disease mechanisms. We find that mice with selective Fmr1 deletion in microglia exhibit audiogenic seizures (AGS) and parvalbumin (PV) neuron loss, recapitulating two key phenotypes observed in FXS (Fmr1^(-/y)) mice. By combining snRNA-Seq...