Department of Biochemistry and Molecular Biology, College of Medicine, Mayo Clinic, Rochester, Minnesota 55905, USA.
J Clin Invest. 2010 Jul;120(7):2537-48. doi: 10.1172/JCI41277.
Somatic activating mutations in Notch1 contribute to the pathogenesis of T cell acute lymphoblastic lymphoma (T-ALL), but how activated Notch1 signaling exerts this oncogenic effect is not completely understood. Here we identify HIV-1 Rev-binding protein (Hrb), a component of the clathrin-mediated endocytosis machinery, as a critical mediator of Notch-induced T-ALL development in mice. Hrb was found to be a direct transcriptional target of Notch1, and Hrb loss reduced the incidence or delayed the onset of T-ALL in mouse models in which activated Notch1 signaling either contributes to or drives leukemogenesis. Consistent with this observation, Hrb supported survival and proliferation of hematopoietic and T cell precursor cells in vitro. We demonstrated that Hrb accelerated the uptake of transferrin, which was required for upregulation of the T cell protooncogene p21. Indeed, iron-deficient mice developed Notch1-induced T-ALL substantially more slowly than control mice, further supporting a critical role for iron uptake during leukemogenesis. Taken together, these results reveal that Hrb is a critical Notch target gene that mediates lymphoblast transformation and disease progression via its ability to satisfy the enhanced demands of transformed lymphoblasts for iron. Further, our data suggest that Hrb may be targeted to improve current treatment or design novel therapies for human T-ALL patients.
Notch1 的体细胞激活突变有助于 T 细胞急性淋巴细胞白血病 (T-ALL) 的发病机制,但 Notch1 信号的激活如何发挥这种致癌作用尚不完全清楚。在这里,我们确定 HIV-1 Rev 结合蛋白 (Hrb),即网格蛋白介导的内吞作用机制的一个组成部分,是 Notch 诱导的小鼠 T-ALL 发展的关键介质。发现 Hrb 是 Notch1 的直接转录靶标,并且 Hrb 的缺失减少了激活的 Notch1 信号在促进或驱动白血病发生的小鼠模型中 T-ALL 的发生率或延迟其发病时间。与这一观察结果一致,Hrb 支持体外造血和 T 细胞前体细胞的存活和增殖。我们证明 Hrb 加速了转铁蛋白的摄取,这对于上调 T 细胞原癌基因 p21 是必需的。事实上,缺铁的小鼠比对照小鼠发展 Notch1 诱导的 T-ALL 要慢得多,这进一步支持了在白血病发生过程中摄取铁的关键作用。总之,这些结果表明 Hrb 是一个关键的 Notch 靶基因,通过满足转化的淋巴母细胞对铁的更高需求,介导淋巴母细胞转化和疾病进展。此外,我们的数据表明,Hrb 可能是一个靶向治疗的靶点,以改善目前治疗或设计新的治疗人类 T-ALL 患者的疗法。