Several preclinical studies on Rett Syndrome (RTT) demonstrated [15,44,45] that CNF1 rescues cognitive deficits, aberrant synaptic plasticity, astrocytes defects, and mitochondrial dysfunction in the MeCP2-308 RTT mouse model through the modulation of brain Rho GTPases that appear to be involved in RTT pathophysiology [29,30,31,32,33]. The gene discussed is MECP2; the disease is Rett syndrome.