Elegant murine experiments have recently shown that the loss of key autophagy genes Atg5 or Atg7 in HSCs or HSPCs leads to a lethal pre-leukemic phenotype in mice, and heterozygous loss of autophagy in a mouse model of AML resulted in more aggressive disease, implicating defects in autophagy in the pathogenesis of MDS and the progression to AML [572,574,577]. This evidence concerns the gene ATG5 and acute myeloid leukemia.