The role of MOZ in the development of the hematopoietic system, spleen and thymus

目前成人白血病的治疗不能令人满意，大多数患者死亡。在过去，大多数癌症的治疗方法都是经验性的，也就是说，一种特定的药物是通过试验和错误而不是合理设计的过程被发现是有效的。为了提高白血病有效治疗的发现率，有必要了解造血是如何调节的，以及可能出错导致癌症的关键点是什么。一些基因在白血病中通常会发生突变。显然，这些基因参与造血调控的某些关键方面。我们正在研究这些基因之一，MOZ，这是突变的急性髓细胞白血病。这项资助的目的是确定该基因的正常功能是什么。白血病最有希望的新疗法之一是直接靶向细胞内基因表达的调节。MOZ是调节造血过程中基因表达和控制不同类型血细胞分化的蛋白质之一。这些新型抗癌药物的可能作用之一是强调MOZ的正常功能。然而，目前我们还不知道MOZ的正常功能是什么，所以不可能测试这个预测。如果我们知道MOZ在控制血液形成的哪些途径中起作用，那么将来可能会利用这些信息以比过去更直接的方式改进目前的抗癌药物。

Current treatment of leukaemia in adults is unsatisfactory with the majority of patients dying. In the past most treatments for cancer have been empirical, that is a particular drug has been found to be effective by trial and error rather than a process of rational design. In order to improve the rate at which effective treatments for leukaemia are found it is necessary to understand how hematopoiesis is regulated and what the critical points are where things can go wrong, leading to cancer. Some genes are commonly found to be mutated in leukaemia. Clearly these genes are involved in some key aspect of regulation of hematopoiesis. We are studying one of these genes, MOZ, which is mutated in acute myeloid leukaemia. The purpose of this grant is to determine what the normal function of this gene is. One of the most promising new treatments for leukaemia is directly targeting the regulation of gene expression inside the cell. MOZ is one of the proteins, which regulates gene expression in hematopoiesis and controls the differentiation of different types of blood cells. One of the possible effects of these new types of anticancer drugs is to accentuate the normal function of MOZ. However, at the moment we don't know what the normal function of MOZ is so it is impossible to test this prediction. If we know which pathways controlling blood formation MOZ is acting in it may be possible, in the future, to use this information to improve on the current anti cancer drugs in a more directed way than has been possible in the past.