We thus aimed to investigate whether targeting the epigenetic regulator DNMT1 using 5-azacytidine (5-AzaC), a hypomethylating agent in clinical use for the treatment of acute myeloid leukemia and myelodysplastic syndromes [7], is efficacious in SHH-MB as well. This evidence concerns the gene DNMT1 and myelodysplastic syndrome.