López-Jiménez Elena, Gómez-López Gonzalo, Leandro-García L Javier, Muñoz Iván, Schiavi Francesca, Montero-Conde Cristina, de Cubas Aguirre A, Ramires Ricardo, Landa Iñigo, Leskelä Susanna, Maliszewska Agnieszka, Inglada-Pérez Lucía, de la Vega Leticia, Rodríguez-Antona Cristina, Letón Rocío, Bernal Carmen, de Campos José M, Diez-Tascón Cristina, Fraga Mario F, Boullosa Cesar, Pisano David G, Opocher Giuseppe, Robledo Mercedes, Cascón Alberto
Hereditary Endocrine Cancer Group, Spanish National Cancer Research Centre, Madrid, Spain.
Mol Endocrinol. 2010 Dec;24(12):2382-91. doi: 10.1210/me.2010-0256. Epub 2010 Oct 27.
The six major genes involved in hereditary susceptibility for pheochromocytoma (PCC)/paraganglioma (PGL) (RET, VHL, NF1, SDHB, SDHC, and SDHD) have been recently integrated into the same neuronal apoptotic pathway where mutations in any of these genes lead to cell death. In this model, prolyl hydroxylase 3 (EglN3) abrogation plays a pivotal role, but the molecular mechanisms underlying its inactivation are currently unknown. The aim of the study was to decipher specific alterations associated with the different genetic classes of PCCs/PGLs. With this purpose, 84 genetically characterized tumors were analyzed by means of transcriptional profiling. The analysis revealed a hypoxia-inducible factor (HIF)-related signature common to succinate dehydrogenase (SDH) and von Hippel-Lindau (VHL) tumors, that differentiated them from RET and neurofibromatosis type 1 cases. Both canonical HIF-1α and HIF-2α target genes were overexpressed in the SDH/VHL cluster, suggesting that a global HIF deregulation accounts for this common profile. Nevertheless, when we compared VHL tumors with SDHB cases, which often exhibit a malignant behavior, we found that HIF-1α target genes showed a predominant activation in the VHL PCCs. Expression data from 67 HIF target genes was sufficient to cluster SDHB and VHL tumors into two different groups, demonstrating different pseudo-hypoxic signatures. In addition, VHL-mutated tumors showed an unexpected overexpression of EglN3 mRNA that did not lead to significantly different EglN3 protein levels. These findings pave the way for more specific therapeutic approaches for malignant PCCs/PGLs management based on the patient's genetic alteration.
最近,参与嗜铬细胞瘤(PCC)/副神经节瘤(PGL)遗传易感性的六个主要基因(RET、VHL、NF1、SDHB、SDHC和SDHD)已被整合到同一神经元凋亡途径中,这些基因中任何一个发生突变都会导致细胞死亡。在这个模型中,脯氨酰羟化酶3(EglN3)的缺失起着关键作用,但其失活的分子机制目前尚不清楚。本研究的目的是解读与不同遗传类型的PCC/PGL相关的特定改变。为此,通过转录谱分析对84个具有明确遗传特征的肿瘤进行了分析。分析揭示了琥珀酸脱氢酶(SDH)和冯·希佩尔-林道(VHL)肿瘤共有的缺氧诱导因子(HIF)相关特征,这使其与RET和1型神经纤维瘤病病例区分开来。经典的HIF-1α和HIF-2α靶基因在SDH/VHL簇中均过度表达,表明整体HIF失调导致了这种共同特征。然而,当我们将VHL肿瘤与通常表现出恶性行为的SDHB病例进行比较时,我们发现HIF-1α靶基因在VHL PCC中表现出主要激活。来自67个HIF靶基因的表达数据足以将SDHB和VHL肿瘤聚类为两个不同的组,表明存在不同的假缺氧特征。此外,VHL突变的肿瘤显示出EglN3 mRNA意外过度表达,但并未导致EglN3蛋白水平有显著差异。这些发现为基于患者基因改变的恶性PCC/PGL管理的更特异性治疗方法铺平了道路。