The ERC research project TREAT-NPM1-AML is focused on improving the therapy of NPM1-mutated AML through the repositioning of old drugs (e.g. actinomycin D) in the light of the genetic lesion and by identifying protein interactors that co-localize with NPM1 (in the nucleus and/or in the cytoplasm) and may represent novel therapeutic targets.
AML
NPM1
FLT3
Genes
Mutations
Cytoplasmic dislocation
AML targeted therapy
Mice models
Prof. Falini and other investigators has broadened the mutational landscape of the AML genome by identifying 23 recurrent mutations.
Generation of novel monoclonal antibodies directed against proteins encoded by mutated genes in lymphomas and leukemias; co-signer of the major classifications (REAL, WHO) of the lympho-hemopoietic neoplasms; immunotherapy and study of the genome landscape of Hodgkin lymphoma; discovery of the NPM1 and BCOR mutations in AML with normal cytogenetics and their translation into clinic; discovery of BRAFV-600E mutation in hairy cell leukemia and development of new molecular targeted therapies of this disease using BRAF inhibitors.