Although in this study BDNF protein dosage was not significantly altered in RTT patients, we report alterations in various MeCP2 and BDNF effectors including LIN28A [40] and several miRNAs (i.e., miR-24, miR-125b, miR-132 [66] and miR-16 [67]) that may underlie some of the pathogenic symptoms in RTT syndrome. This evidence concerns the gene BDNF and Rett syndrome.