Research ArticlesMedicine

Integration of Hedgehog and mutant FLT3 signaling in myeloid leukemia

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Science Translational Medicine  10 Jun 2015:
Vol. 7, Issue 291, pp. 291ra96
DOI: 10.1126/scitranslmed.aaa5731

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Hedgehog to the rescue

Acute myeloid leukemia is generally difficult to treat, and the presence of internal tandem duplication in a gene called FLT3 (FLT3-ITD) is associated with a particularly poor prognosis. Lim et al. discovered that patients with FLT3-ITD leukemia also have increased activity of the Hedgehog protein signaling pathway. Experiments in mouse models of FLT3-ITD confirmed the functional role of Hedgehog in the development of leukemia and showed that combined treatment targeting FLT3 and Hedgehog is effective as a therapeutic strategy in this setting.