Calabresi Laura, Balliu Manjola, Bartalucci Niccolò
Center for Research and Innovation for Myeloproliferative Neoplasms, Department of Experimental and Clinical Medicine, University of Florence, Florence, Italy.
Center for Research and Innovation for Myeloproliferative Neoplasms, Department of Experimental and Clinical Medicine, University of Florence, Florence, Italy.
Methods Cell Biol. 2022;171:81-109. doi: 10.1016/bs.mcb.2022.04.005. Epub 2022 Jul 26.
Philadelphia-negative myeloproliferative neoplasms (pH-MPNs) origin from the clonal expansion of hematopoietic stem cells with acquired mutations leading to uncontrolled proliferation of differentiated myeloid cells. The main entities of Ph-MPNs are represented by Polycythemia Vera (PV), Essential Thrombocythemia (ET) and Myelofibrosis (MF) that are characterized by microvascular disorders, thrombosis and bleeding, splenomegaly secondary to extramedullary hematopoiesis, various degree of bone marrow fibrosis and a progressive risk of leukemic transformation. Somatic mutations in myeloid genes including JAK2, CALR, and MPL cause the constitutive activation of the Janus Kinase 2 (JAK)/signal transducer and activator of transcription (STAT) pathway that confers proliferative and differentiative advantage to mutated hematopoietic progenitors and ultimately drives the development of a Ph-MPNs phenotype. Beyond the JAK/STAT axis, a wide number of intracellular signaling pathways were found deregulated in Ph-MPNs including the phosphatidylinositol-3-kinase (PI3K)/protein kinase B (Akt)/mammalian target of rapamycin (mTOR) constitutive activation. In this chapter, we provide a detailed protocol for the immunoblotting assisted assessment of Ph-MPNs pathways activation. This protocol can be easily adapted to study protein expression and phosphorylation of hematopoietic stem progenitors and differentiated cell lineages.
费城染色体阴性骨髓增殖性肿瘤(pH-MPNs)起源于造血干细胞的克隆性扩增,伴有获得性突变,导致分化的髓系细胞不受控制地增殖。pH-MPNs的主要类型包括真性红细胞增多症(PV)、原发性血小板增多症(ET)和骨髓纤维化(MF),其特征为微血管疾病、血栓形成和出血、髓外造血继发脾肿大、不同程度的骨髓纤维化以及白血病转化的渐进性风险。包括JAK2、CALR和MPL在内的髓系基因的体细胞突变导致Janus激酶2(JAK)/信号转导子和转录激活子(STAT)途径的组成性激活,赋予突变的造血祖细胞增殖和分化优势,并最终驱动pH-MPNs表型的发展。除了JAK/STAT轴外,还发现许多细胞内信号通路在pH-MPNs中失调,包括磷脂酰肌醇-3-激酶(PI3K)/蛋白激酶B(Akt)/雷帕霉素哺乳动物靶蛋白(mTOR)的组成性激活。在本章中,我们提供了一份详细的免疫印迹辅助评估pH-MPNs途径激活的方案。该方案可轻松适用于研究造血干祖细胞和分化细胞谱系的蛋白质表达和磷酸化。