DYSREGULATED NFKB PATHWAY SIGNALING IN MYELOPROLIFERATIVE NEOPLASMS

7.项目摘要/摘要 骨髓增生性肿瘤(MPN)是一种慢性血液疾病，可导致严重的症状和 早逝。最近出现了新的治疗方法，可以帮助缓解症状，但它们不起作用 可靠地减缓或阻止疾病的进展。我们试图更好地了解是什么推动了疾病的发展和 MPN的进展，以便我们可以为这些疾病的患者开发更好的治疗方法。 我们的初步数据表明，一条称为NFkB通路的信号通路在 MPN。我们假设这一途径有助于MPN的发展和进展。因此， 我们已经提出了一项结合小鼠和人类的研究来确定NFkB途径是如何 有助于MPN的发病，并评估抑制NFkB信号是否具有潜在的作用 对MPN患者的治疗益处。

7. Project Summary/Abstract Myeloproliferative neoplasms (MPNs) are chronic blood disorders that that can cause severe symptoms and early death. New treatments have become available recently that help ameliorate symptoms, but they do not reliably slow or halt disease progression. We seek to better understand what drives disease development and progression in MPNs, so that we can develop better therapies for patients with these diseases. Our preliminary data indicates that a signaling pathway called the NFkB pathway is abnormally activated in MPNs. We hypothesize that this pathway contributes to the development and progression of MPNs. Therefore, we have proposed a combination of mouse and human studies to determine how the NFkB pathway contributes to MPN pathogenesis, and to evaluate whether inhibition of NFkB signaling may have potential therapeutic benefits for MPN patients.