Hh/Gli antagonist in acute myeloid leukemia with CBFA2T3-GLIS2 fusion gene

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• 作者：Riccardo Masetti ; Salvatore Nicola Bertuccio…
• 关键词：Acute myeloid leukemia ; Acute megakaryoblastic leukemia ; CBFA2T3 ; GLIS2 ; GANT61 ; Hedgehog pathway
• 刊名：Journal of Hematology & Oncology
• 出版年：2017
• 出版时间：December 2017
• 年：2017
• 卷：10
• 期：1
• 全文大小：1183KB
• 刊物主题：Oncology; Hematology; Cancer Research;
• 出版者：BioMed Central
• ISSN：1756-8722
• 卷排序：10

文摘

BackgroundCBFA2T3-GLIS2 is a fusion gene found in 17% of non-Down syndrome acute megakaryoblastic leukemia (non-DS AMKL, FAB M7) and in 8% of pediatric cytogenetically normal acute myeloid leukemia (CN-AML, in association with several French-American-British (FAB) subtypes). Children with AML harboring this aberration have a poor outcome, regardless of the FAB subtype. This fusion gene drives a peculiar expression pattern and leads to overexpression of some of Hedgehog-related genes. GLI-similar protein 2 (GLIS2) is closely related to the GLI family, the final effectors of classic Hedgehog pathway. These observations lend compelling support to the application of GLI inhibitors in the treatment of AML with the aberration CBFA2T3-GLIS2. GANT61 is, nowadays, the most potent inhibitor of GLI family proteins.