Cancer
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Pathogenesis of ASXL1-mutated Acute Myeloid Leukemia (AML): deciphering mechanisms for novel rational interventions

Institution: Clinic for Haematology, Oncology, and Tumor immunology, Charité Universitätsmedizin Berlin
Applicant: Dr. Francis Baumgartner
Funding line:
First and Second Applications
Image
Snapshot: Using the Biomek Laboratory Automation Workstation for parallel processing of 96-well plates.
(© Charité - Universitätsmedizin Berlin Dr. med. F. Baumgartner)

This project aims to deepen the molecular understanding of ASXL1-mutated (ASXL1mut) acute myeloid leukemia (AML) and subsequently leverage the findings to improve only very limited therapeutic possibilities. ASXL1mut frequently occur in myeloid diseases and, especially in AML, are accompanied by an unfavorable prognosis. By combining Asxl1mut with a genetic system randomly causing mutations, leukemia occurs in mice. Arising Asxl1mut AML are then sequenced and compared to human ASXL1mut AML, enabling the pinpointing of novel crucial events in leukemic development. The most promising identified cancer genes are then (pre)clinically validated for their therapeutic use, e.g., through the development of biomarkers or individualized targeted (combination) therapies.