Department of Internal Medicine I, Ulm University Hospital, Ulm, Germany.
Department of Internal Medicine I, Ulm University Hospital, Ulm, Germany; Department of Urology, Ulm University, Ulm, Germany.
Cell Stem Cell. 2021 Jun 3;28(6):1105-1124.e19. doi: 10.1016/j.stem.2021.03.005. Epub 2021 Apr 28.
Personalized in vitro models for dysplasia and carcinogenesis in the pancreas have been constrained by insufficient differentiation of human pluripotent stem cells (hPSCs) into the exocrine pancreatic lineage. Here, we differentiate hPSCs into pancreatic duct-like organoids (PDLOs) with morphological, transcriptional, proteomic, and functional characteristics of human pancreatic ducts, further maturing upon transplantation into mice. PDLOs are generated from hPSCs inducibly expressing oncogenic GNAS, KRAS, or KRAS with genetic covariance of lost CDKN2A and from induced hPSCs derived from a McCune-Albright patient. Each oncogene causes a specific growth, structural, and molecular phenotype in vitro. While transplanted PDLOs with oncogenic KRAS alone form heterogenous dysplastic lesions or cancer, KRAS with CDKN2A loss develop dedifferentiated pancreatic ductal adenocarcinomas. In contrast, transplanted PDLOs with mutant GNAS lead to intraductal papillary mucinous neoplasia-like structures. Conclusively, PDLOs enable in vitro and in vivo studies of pancreatic plasticity, dysplasia, and cancer formation from a genetically defined background.
个性化体外胰腺发育不良和癌变模型一直受到人类多能干细胞(hPSC)向外分泌胰腺谱系分化不足的限制。在这里,我们将 hPSC 分化为具有人胰腺导管形态、转录组、蛋白质组和功能特征的胰腺导管样类器官(PDLO),进一步在移植到小鼠后成熟。PDLO 可从诱导表达致癌基因 GNAS、KRAS 或 KRAS 的 hPSC 中产生,也可从源自 McCune-Albright 患者的诱导 hPSC 中产生,这些 hPSC 具有缺失 CDKN2A 的遗传协方差。每个致癌基因在体外引起特定的生长、结构和分子表型。虽然单独携带致癌 KRAS 的移植 PDLO 形成异质性发育不良病变或癌症,但 KRAS 与 CDKN2A 缺失会导致去分化的胰腺导管腺癌。相比之下,携带突变 GNAS 的移植 PDLO 会导致类似于导管内乳头状黏液性肿瘤的结构。总之,PDLO 可用于从遗传定义的背景下进行胰腺可塑性、发育不良和癌症形成的体外和体内研究。