Revealing vulnerabilities in early hematopoietic stages to identify novel therapeutic targets in pediatric non-DS AMKL
Deadline: 20/03/2026
Scientific-Disciplinary Group
06/MEDS-20 - Paediatrics And Child Neuropsychiatry
Description
This project will investigate therapeutic vulnerabilities in NUP98::NSD1- and KDM5A-driven AML using an integrated preclinical strategy. AML cell lines and primary ex vivo samples will be used to test venetoclax–azacitidine–based regimens combined with menin inhibitors, evaluating drug sensitivity, apoptosis, and synergistic effects on leukemic cell survival and self-renewal. To enhance translational relevance, in vitro studies will incorporate a 3D bone marrow niche–mimicking model. Results will guide in vivo experiments in immunodeficient mice engrafted with primary AML cells, conducted in collaboration with the Padova laboratory. Patient-derived xenografts will be randomized to receive single-agent or combination treatments to assess therapeutic efficacy, synergy, and overall survival. Longitudinal monitoring and post-treatment molecular analyses will provide insights into mechanisms of response and resistance.
Job posting website
Funding body
ALMA MATER STUDIORUM - UNIVERSITA' DI BOLOGNA - - DIPARTIMENTO DI SCIENZE MEDICHE E CHIRURGICHE
How to apply
Other
Selection process
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