Therefore, the transcriptome and molecular readouts are an ideal outcome to assess therapeutic benefit for MECP2-related disorders, and genes that are modulated by acute changes in MeCP2 levels may help us discover expression programs that are either involved in driving pathogenesis when MeCP2 is depleted in healthy neurons or responsive in reversal of pathogenesis when MeCP2 levels are normalized in MDS neurons. Here, MECP2 is linked to myelodysplastic syndrome.