Vu Hong Nhung, Radjasandirane Ragousandirane, Diharce Julien, de Brevern Alexandre G
Université Paris Cité and Université de la Réunion, INSERM, EFS, BIGR U1134, DSIMB Bioinformatics Team, F-75015 Paris, France.
Université Paris Cité and Université de la Réunion, INSERM, EFS, BIGR U1134, DSIMB Bioinformatics Team, F-97715 Saint Denis Messag, France.
Int J Mol Sci. 2025 Apr 15;26(8):3727. doi: 10.3390/ijms26083727.
Janus kinase 2 (JAK2) is an important intracellular mediator of cytokine signaling. Mutations in the JAK2 gene are associated with myeloproliferative neoplasms (MPNs) such as polycythemia vera (PV) and essential thrombocythemia (ET), while aberrant JAK2 activity is also associated with a number of immune diseases. The acquired somatic mutation JAK2 V617F (95% of cases of PV and in 55-60% of cases of ET), which constitutively activates the JAK2, is the most common molecular event in MPN. The development of specific JAK2 inhibitors is therefore of considerable clinical importance. Ruxolitinib is a JAK inhibitor recently approved by the FDA/EMA and effective in relieving symptoms in patients with MPN. Ruxolitinib binds to the JAK2 last domain, namely JH1; its action on the dynamics of the domain is still only partially known. Using Molecular Dynamics simulations, we have analyzed the JH1 domain in four different states as follows: (i) alone, (ii) with one phosphorylation, (iii) adding Ruxolitinib, and (iv) with five phosphorylations and Ruxolitinib. The ligand induces a dynamic behavior similar to the inactive form of JH1, with a less flexible state than the phosphorylated active form of JH1. This study highlights the inhibitory effect of Ruxolitinib on the JH1 domain, demonstrating the importance of dynamics in regulating JH1 activation.
Janus激酶2(JAK2)是细胞因子信号传导的重要细胞内介质。JAK2基因突变与骨髓增殖性肿瘤(MPN)相关,如真性红细胞增多症(PV)和原发性血小板增多症(ET),而异常的JAK2活性也与多种免疫疾病有关。获得性体细胞突变JAK2 V617F(95%的PV病例和55 - 60%的ET病例)可组成性激活JAK2,是MPN中最常见的分子事件。因此,开发特异性JAK2抑制剂具有相当重要的临床意义。芦可替尼是一种最近被美国食品药品监督管理局/欧洲药品管理局批准的JAK抑制剂,对缓解MPN患者的症状有效。芦可替尼与JAK2的最后一个结构域即JH1结合;其对该结构域动力学的作用仍仅部分为人所知。我们使用分子动力学模拟,分析了JH1结构域在四种不同状态下的情况:(i)单独存在,(ii)有一个磷酸化位点,(iii)添加芦可替尼,以及(iv)有五个磷酸化位点且添加芦可替尼。该配体诱导出一种类似于JH1非活性形式的动态行为,其状态比JH1磷酸化活性形式的状态更不灵活。这项研究突出了芦可替尼对JH1结构域的抑制作用,证明了动力学在调节JH1激活中的重要性。