GATA1-related leukaemias

Nat Rev Cancer. 2008 Apr;8(4):279-87. doi: 10.1038/nrc2348.


GATA1 is a prototypical lineage-restricted transcription factor that is central to the correct differentiation, proliferation and apoptosis of erythroid and megakaryocytic cells. Mutations in GATA1 can generate a truncated protein, which contributes to the genesis of transient myeloproliferative disorder (TMD) and acute megakaryoblastic leukaemia (AMKL) in infants with Down syndrome. Similarly, Gata1 knockdown to 5% of its wild-type level causes high incidence of erythroid leukaemia in mice. The GATA1-related leukaemias in both human and mouse could provide important insights into the mechanism of multi-step leukaemogenesis. Efforts are afoot to produce mouse models that are reflective of TMD and AMKL.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Disease Progression
  • GATA1 Transcription Factor / physiology*
  • Humans
  • Leukemia, Megakaryoblastic, Acute / metabolism*
  • Mice
  • Myeloproliferative Disorders / metabolism*


  • GATA1 Transcription Factor