Indeed, leukemic progression in FA cells is associated with two main events: genetic inactivation (translocations, deletions, or mutations) of the RUNX1/AML1 locus [56], a master regulator of the differentiation program of the cells, and amplification of the 1q/MDM4 locus, which leads to overexpression of the p53 suppressor MDM4, allowing downregulation of basal p53 activation without the need for inactivating mutations in the tumor suppressor [57]. The gene discussed is RUNX1; the disease is Friedreich ataxia.