Laboratoire de Biologie et Pharmacologie Appliquée, Centre Nationale de la Recherche, Unité Mixte de Recherche 8113, Ecole Normale Supérieure de Cachan, Cachan, France;
Laboratoire de Biologie et Pharmacologie Appliquée, Centre Nationale de la Recherche, Unité Mixte de Recherche 8113, Ecole Normale Supérieure de Cachan, Cachan, France; Department of Internal Medicine I, Division of Hematology and Hemostaseology, and.
Blood. 2014 Jul 3;124(1):111-20. doi: 10.1182/blood-2013-10-534685. Epub 2014 Mar 27.
In systemic mastocytosis (SM), clinical problems arise from factor-independent proliferation of mast cells (MCs) and the increased release of mediators by MCs, but no human cell line model for studying MC activation in the context of SM is available. We have created a stable stem cell factor (SCF) -dependent human MC line, ROSA(KIT WT), expressing a fully functional immunoglobulin E (IgE) receptor. Transfection with KIT D816V converted ROSA(KIT WT) cells into an SCF-independent clone, ROSA(KIT D816V), which produced a mastocytosis-like disease in NSG mice. Although several signaling pathways were activated, ROSA(KIT D816V) did not exhibit an increased, but did exhibit a decreased responsiveness to IgE-dependent stimuli. Moreover, NSG mice bearing ROSA(KIT D816V)-derived tumors did not show mediator-related symptoms, and KIT D816V-positive MCs obtained from patients with SM did not show increased IgE-dependent histamine release or CD63 upregulation. Our data show that KIT D816V is a disease-propagating oncoprotein, but it does not activate MCs to release proinflammatory mediators, which may explain why mediator-related symptoms in SM occur preferentially in the context of a coexisting allergy. ROSA(KIT D816V) may provide a valuable tool for studying the pathogenesis of mastocytosis and should facilitate the development of novel drugs for treating SM patients.
在系统性肥大细胞增多症 (SM) 中,临床问题源于肥大细胞 (MC) 的无因增殖和 MC 中介质的过度释放,但目前尚无可用于研究 SM 背景下 MC 激活的人类细胞系模型。我们创建了一种稳定的干细胞因子 (SCF) 依赖性人类 MC 系 ROSA(KIT WT),其表达功能完整的免疫球蛋白 E (IgE) 受体。转染 KIT D816V 将 ROSA(KIT WT) 细胞转化为一种 SCF 非依赖性克隆体 ROSA(KIT D816V),该克隆体在 NSG 小鼠中产生了类似于肥大细胞增多症的疾病。尽管几种信号通路被激活,但 ROSA(KIT D816V) 并未表现出增强的反应性,而是表现出降低的 IgE 依赖性刺激反应性。此外,携带 ROSA(KIT D816V)衍生肿瘤的 NSG 小鼠未表现出与介质相关的症状,并且从 SM 患者中获得的 KIT D816V 阳性 MC 未显示出增加的 IgE 依赖性组胺释放或 CD63 上调。我们的数据表明,KIT D816V 是一种促进疾病进展的致癌蛋白,但它不会激活 MC 释放促炎介质,这可能解释了为什么 SM 中的介质相关症状优先出现在共存过敏的情况下。ROSA(KIT D816V) 可能为研究肥大细胞增多症的发病机制提供有价值的工具,并应有助于开发治疗 SM 患者的新型药物。