BH3 mimetic therapies for CBFA2T3::GLIS2 pediatric acute megakaryoblastic leukemia.

IF 13.8 1区医学 Q1 BIOCHEMISTRY & MOLECULAR BIOLOGY

Trends in molecular medicine Pub Date : 2025-10-06 DOI:10.1016/j.molmed.2025.09.006

Caroline Capdevielle, Sarah St-Amand, Mathieu Neault, Tanja A Gruber, Sonia Cellot, Frédérick A Mallette

引用次数: 0

Abstract

Pediatric acute megakaryoblastic leukemia (AMKL) associated with the CBFA2T3::GLIS2 (CG2) gene fusion is distinctive by virtue of its aggressiveness and ability to transform normal cells in a single event. The dismal therapeutic response of children to this disease has stimulated comprehensive studies on the molecular mechanism of CG2-driven tumorigenesis. In this Opinion, we highlight various investigative strategies, used in parallel by multiple independent research teams, that point to a specific dependence of CG2-expressing leukemias on the B cell leukemia/lymphoma-2 (BCL-2) family of antiapoptotic proteins. We propose that this intrinsic feature renders these leukemias particularly vulnerable to BCL-2 homology 3 (BH3) mimetics.

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BH3模拟疗法治疗CBFA2T3::GLIS2儿童急性巨核细胞白血病。

与CBFA2T3::GLIS2 （CG2）基因融合相关的儿童急性巨核母细胞白血病（AMKL）的独特之处在于其侵袭性和在一次事件中转化正常细胞的能力。儿童对这种疾病的治疗反应不佳，刺激了对cg2驱动的肿瘤发生的分子机制的全面研究。在本意见中，我们强调了多个独立研究团队并行使用的各种调查策略，这些策略指出表达cg2的白血病对B细胞白血病/淋巴瘤-2 （BCL-2）抗凋亡蛋白家族的特异性依赖。我们认为这种内在特征使得这些白血病特别容易受到BCL-2同源3 （BH3）模拟物的影响。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Trends in molecular medicine 医学-生化与分子生物学

CiteScore

24.60

自引率

0.00%

发文量

142

审稿时长

6-12 weeks

期刊介绍： Trends in Molecular Medicine (TMM) aims to offer concise and contextualized perspectives on the latest research advancing biomedical science toward better diagnosis, treatment, and prevention of human diseases. It focuses on research at the intersection of basic biology and clinical research, covering new concepts in human biology and pathology with clear implications for diagnostics and therapy. TMM reviews bridge the gap between bench and bedside, discussing research from preclinical studies to patient-enrolled trials. The major themes include disease mechanisms, tools and technologies, diagnostics, and therapeutics, with a preference for articles relevant to multiple themes. TMM serves as a platform for discussion, pushing traditional boundaries and fostering collaboration between scientists and clinicians. The journal seeks to publish provocative and authoritative articles that are also accessible to a broad audience, inspiring new directions in molecular medicine to enhance human health.