Partial FMRP expression is sufficient to normalize neuronal hyperactivity in Fragile X neurons

Fragile X syndrome (FXS) is the most common genetic form of intellectual disability caused by a CGG repeat expansion in the 5′‐UTR of the Fragile X mental retardation gene FMR1 , triggering epigenetic silencing and the subsequent absence of the protein, FMRP . Reactivation of FMR1 represents an attractive therapeutic strategy targeting the genetic root cause of FXS. However, largely missing in the FXS field is an understanding of how much FMR1...