Overcoming the differentiation block in acute myeloid leukaemia

急性髓系白血病(AML)是一种总存活率较低的侵袭性白血病。大约50%的AML病例具有使PU1失效的基因突变，而PU1又会改变导致白血病的许多其他基因的表达。我们已经开发了新的AML模型，可以可逆地抑制PU.1，并表明重新启动PU.1功能会导致AML回归。该项目旨在了解PU1在AML中的功能，并确定治疗耐药疾病的合理药物靶点。

Acute myeloid leukaemia (AML) is an aggressive leukaemia with poor overall survival. About 50% of AML cases have genetic mutations that disable PU.1, which in turn alters the expression of many other genes that cause leukaemia. We have developed new AML models allowing reversible inhibition of PU.1, and have shown that re-engaging PU.1 function causes AML regression. This project aims to understand PU.1 functions in AML and identify rational drug targets for treatment-resistant disease.