Abstract

How to unmask unique vulnerabilities in leukaemia Acute myeloid leukaemia (AML) is a group of haematologic malignancies that have been traditionally difficult to classify and treat. Nucleophosmin (NPM1) mutations are the most frequent genetic alteration (about 30 per cent) in AML and NPM1-mutated AML is a new entity in the WHO classification of myeloid neoplasms. However, mechanisms of leukemogenesis and a specific therapy for this leukaemia are missing. The ContraNPM1AML project aims to unravel the complex network of molecular interactions that take place in this distinct genetic subtype and find their vulnerabilities to identify new targets for therapy. The expected discoveries will lead to novel therapeutic approaches and make clinical trials available to patients.

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