Acute megakaryoblastic leukemia future or investigational therapies

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Non-Down syndrome AMKL:

  • GANT61— a Hedgehog HH/GLI antagonist causes apoptosis of the AML cell lines by targeting the CBFA2T3-GLIS2 fusion gene (expressed in 17% of non-Down syndrome patients with AMKL).[2]

Down syndrome and non-DS AMKL:

References

  1. Schweitzer, Jana; Zimmermann, Martin; Rasche, Mareike; von Neuhoff, Christine; Creutzig, Ursula; Dworzak, Michael; Reinhardt, Dirk; Klusmann, Jan-Henning (2015). "Improved outcome of pediatric patients with acute megakaryoblastic leukemia in the AML-BFM 04 trial". Annals of Hematology. 94 (8): 1327–1336. doi:10.1007/s00277-015-2383-2. ISSN 0939-5555.
  2. Masetti, Riccardo; Bertuccio, Salvatore Nicola; Astolfi, Annalisa; Chiarini, Francesca; Lonetti, Annalisa; Indio, Valentina; De Luca, Matilde; Bandini, Jessica; Serravalle, Salvatore; Franzoni, Monica; Pigazzi, Martina; Martelli, Alberto Maria; Basso, Giuseppe; Locatelli, Franco; Pession, Andrea (2017). "Hh/Gli antagonist in acute myeloid leukemia with CBFA2T3-GLIS2 fusion gene". Journal of Hematology & Oncology. 10 (1). doi:10.1186/s13045-017-0396-0. ISSN 1756-8722.
  3. Liu, Ting; Liu, Xin; Li, Wenhua (2016). "Tetrandrine, a Chinese plant-derived alkaloid, is a potential candidate for cancer chemotherapy". Oncotarget. 7 (26): 40800–40815. doi:10.18632/oncotarget.8315. ISSN 1949-2553.

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