GATA1-related leukaemias

Ritsuko Shimizu, James Douglas Engel, Masayuki Yamamoto

Research output: Contribution to journalReview articlepeer-review

77 Citations (Scopus)


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.

Original languageEnglish
Pages (from-to)279-287
Number of pages9
JournalNature Reviews Cancer
Issue number4
Publication statusPublished - 2008 Apr

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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