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Leuk Res. 2011 Feb;35(2):214-8. doi: 10.1016/j.leukres.2010.07.013. Epub 2010 Aug 6.

Biologic activity of triptolide in t(8;21) acute myeloid leukemia cells.

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Laboratory of Molecular Carcinogenesis and Targeted Therapy for Cancer, Guangzhou Institute of Biomedicine and Health & State Key Laboratory of Biomembrane and Membrane Biotechnology, Institute of Zoology, Chinese Academy of Sciences, China.


Triptolide is a compound isolated from the traditional Chinese medicinal herb Tripterygium wilfordii that shows potent anti-tumor activities, but its effects on acute myeloid leukemia with t(8;21) remain unclear. Here we report that triptolide inhibits cell proliferation and induces apoptosis in a dose- and time-dependent manner of t(8;21)-bearing Kasumi-1, SKNO-1 and CD34+ cells harvested from bone marrow samples of patients with t(8;21) leukemia. We show that triptolide triggers cleavage of the resultant AML1-ETO fusion protein of t(8;21), and causes downregulation of C-KIT followed by inhibition of JAK-STAT signaling. Triptolide downregulates p65 and inhibits the DNA-binding activity of NF-κB. Our data indicate that triptolide might be an effective agent for t(8;21) leukemia.

[Indexed for MEDLINE]

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