To assess whether pharmacological interventions targeting Rho GTPases may be an effective therapeutic strategy for RTT, we evaluated the effects of a single icv injection of CNF1 in MeCP2-308 hemizygous (hz) male mice, a mouse model of RTT that expresses a truncated form of the MeCP2 gene [74,75,76,77]. This evidence concerns the gene MECP2 and Rett syndrome.