In RTT mouse models, transgenic expression of either Mecp2 isoform can rescue RTT phenotypes to different extents [14,15]. However, gene therapy delivery of MECP2 into the affected cells or drug therapies to induce MECP2 expression has to be carried out with caution, as even mild overexpression of MeCP2 can lead to progressive neurological disorders [16,17]. Currently, limited knowledge exists on MECP2/Mecp2 regulation, with no specific knowledge on possible differential MECP2/Mecp2 isoform-specific regulatory mechanisms.
