Gastroenteropancreatic neuroendocrine neoplasms: genes, therapies and models.
Gastroenteropancreatic neuroendocrine neoplasms: genes, therapies and models.
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DOI：
10.1242/dmm.029595
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发表时间：
2018-02-26
影响因子：
4.3
通讯作者：
Sato T
中科院分区：
文献类型：
--
作者：
Kawasaki K;Fujii M;Sato T
胃肠胰神经内分泌肿瘤（GEP-NENs）是指一组具有神经内分泌细胞表型的异质性癌症，主要分为两种亚型之一：胃肠胰神经内分泌肿瘤（GEP-NETs；高分化）或胃肠胰神经内分泌癌（GEP-NECs；低分化）。尽管最初被定义为孤儿癌症，但其发病率稳步上升凸显了更好地了解其病因的必要性。积累的流行病学和临床数据揭示了这些疾病的病理特征。然而，患者数量相对较少阻碍了大规模临床试验的进行，从而阻碍了新治疗策略的开发。为了克服这一限制，需要忠实反映这些疾病临床特征的可治疗疾病模型。在这篇综述中，我们基于传统疾病模型，如基因工程小鼠模型（GEMM）和细胞系，总结了目前对这些疾病的遗传学和生物学的理解，并讨论了模型与受影响人类之间的表型差异。我们还重点介绍了源自人类临床样本的新兴疾病模型，包括患者来源的异种移植模型和类器官，它们可能为 GEP-NEN 提供生物学和治疗见解。摘要：胃肠胰神经内分泌肿瘤一直是一个研究不足的实体，治疗选择有限。我们讨论疾病模型，包括新兴的类器官，它们可以为这些疾病提供生物学和治疗方面的见解。
Gastroenteropancreatic neuroendocrine neoplasms (GEP-NENs) refer to a group of heterogeneous cancers of neuroendocrine cell phenotype that mainly fall into one of two subtypes: gastroenteropancreatic neuroendocrine tumors (GEP-NETs; well differentiated) or gastroenteropancreatic neuroendocrine carcinomas (GEP-NECs; poorly differentiated). Although originally defined as orphan cancers, their steadily increasing incidence highlights the need to better understand their etiology. Accumulating epidemiological and clinical data have shed light on the pathological characteristics of these diseases. However, the relatively low number of patients has hampered conducting large-scale clinical trials and hence the development of novel treatment strategies. To overcome this limitation, tractable disease models that faithfully reflect clinical features of these diseases are needed. In this Review, we summarize the current understanding of the genetics and biology of these diseases based on conventional disease models, such as genetically engineered mouse models (GEMMs) and cell lines, and discuss the phenotypic differences between the models and affected humans. We also highlight the emerging disease models derived from human clinical samples, including patient-derived xenograft models and organoids, which may provide biological and therapeutic insights into GEP-NENs. Summary: Gastroenteropancreatic neuroendocrine neoplasms have been an understudied entity with limited treatment options. We discuss the disease models, including emerging organoids, that may provide biological and therapeutic insight into these diseases.
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影响因子：
8.8
作者：
Couvelard, A;O'Toole, D;Turley, H;Leek, R;Sauvanet, A;Degott, C;Ruszniewski, P;Belghiti, J;Harris, AL;Gatter, K;Pezzella, F
通讯作者：
Pezzella, F
DOI：
10.1016/j.neo.2016.08.003
发表时间：
2016-10
期刊：
Neoplasia (New York, N.Y.)
影响因子：
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作者：
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通讯作者：
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作者：
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通讯作者：
Hanahan, D
影响因子：
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作者：
Calvete, Oriol;Reyes, Jose;Benitez, Javier
通讯作者：
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影响因子：
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作者：
Bertolino, P;Tong, WM;Zhang, CX
通讯作者：
Zhang, CX