Treatment of primary MDS specimens resulted in marked upregulation of GATA1 (P = 0.0159) (Figure 6A), accompanied by expansion of erythroid precursors (Figure 6B) and increased expression of the CD71 transferrin receptor (P = 0.0338) (Figure 6C), indicating reversal of myeloid lineage skewing. Here, GATA1 is linked to myelodysplastic syndrome.