Bakiri Latifa, Wagner Erwin F
Laboratory Genes and Disease, Department of Laboratory Medicine, Medical University of Vienna (MUW), Vienna, Austria.
Laboratory Genes and Disease, Department of Dermatology, Medical University of Vienna (MUW), Vienna, Austria.
Cell Cycle. 2024 May-Jun;23(9-12):834-842. doi: 10.1080/15384101.2024.2429968. Epub 2024 Nov 24.
Hepatocellular carcinoma (HCC), a leading cause of cancer-related death with limited therapies, is a complex disease developing in a background of Hepatitis Virus infection or systemic conditions, such as the metabolic syndrome. Investigating HCC pathogenesis in model organisms is therefore crucial for developing novel diagnostic and therapeutic tools. Genetically engineered mouse models (GEMMs) have been instrumental in recapitulating the local and systemic features of HCC. Early studies using GEMMs and patient material implicated members of the dimeric Activator Protein-1 (AP-1) transcription factor family, such as c-Jun and c-Fos, in HCC formation. In a recent report, we described how switchable, hepatocyte-restricted expression of a single-chain c-Jun~Fra-2 protein, functionally mimicking the c-Jun/Fra-2 AP-1 dimer, results in spontaneous and largely reversible liver tumors in GEMMs. Dysregulated cell cycle, inflammation, and dyslipidemia are observed at early stages and tumors display molecular HCC signatures. We demonstrate that increased c-Myc expression is an essential molecular determinant of tumor formation that can be therapeutically targeted using the BET inhibitor JQ1. Here, we discuss these findings with additional results illustrating how AP-1 GEMMs can foster preclinical research on liver diseases with novel perspectives offered by the constantly increasing wealth of HCC-related datasets.
肝细胞癌(HCC)是癌症相关死亡的主要原因之一,治疗手段有限,是一种在肝炎病毒感染或全身性疾病(如代谢综合征)背景下发展起来的复杂疾病。因此,在模式生物中研究HCC发病机制对于开发新的诊断和治疗工具至关重要。基因工程小鼠模型(GEMMs)有助于重现HCC的局部和全身特征。早期使用GEMMs和患者材料的研究表明,二聚体激活蛋白-1(AP-1)转录因子家族成员,如c-Jun和c-Fos,参与了HCC的形成。在最近的一份报告中,我们描述了单链c-Jun~Fra-2蛋白的可切换、肝细胞特异性表达如何在功能上模拟c-Jun/Fra-2 AP-1二聚体,从而在GEMMs中导致自发性且大多可逆的肝肿瘤。在早期阶段观察到细胞周期失调、炎症和血脂异常,肿瘤表现出分子HCC特征。我们证明,c-Myc表达增加是肿瘤形成的一个关键分子决定因素,可使用BET抑制剂JQ1进行治疗靶向。在此,我们讨论这些发现以及其他结果,这些结果说明了AP-1 GEMMs如何能够以不断增加的大量HCC相关数据集提供的新视角促进肝脏疾病的临床前研究。