Global brain transduction of the instability-prone Mecp2 transgene by systemic AAV-PHP.eB administration is safe and effective in protecting male and female Mecp2 mutant mice from the Rett syndrome disease phenotype.

Mecp2 expression is cell cycle-dependent and negatively regulates quiescence exit.

In mouse models of Rett Syndrome, global loss of the Mecp2 gene induces microglia to engulf excess synapses, while microglia-specific loss or gain of Mecp2 has little impact on disease.

Integrated analysis of transcriptional profiles from mice carrying distinct Mecp2 mutant alleles revealed that MeCP2 regulates Gdf11 expression in the brain, and that Gdf11 is a dosage-sensitive gene whose levels impact neuronal function and animal behavior.

A novel comparative genomics framework identifies MECP2 network proteins targeted by existing drugs, with three drugs validated in an in vitro Rett syndrome model.

Excessive inflammation of the brain in early life predisposes individuals to neurodevelopmental disorders by preventing synapses from developing correctly.

Single molecular imaging of MeCP2 at high spatial/temporal resolution identifies distinct structural domains contributing to its dynamic behaviors and chromatin interactions in live neurons.

Transgenic mice with Rett-causing mutations in MeCP2 reveal that a basic cluster in the C-terminus of the protein binds DNA and that both the methyl-CpG binding domain and the transcriptional repression domain are necessary to elicit toxicity in MECP2 duplication syndrome.

Individuals with deletions or duplications of the NUDT21 gene have altered levels of MeCP2 protein and intellectual disabilities.

Excitatory signaling impairment contributes to neurological deficits shared by Rett syndrome and a number of postnatal neuropsychiatric disorders.