Highlighted Publications

Ontogeny Dictates Oncogenic Potential, Lineage Hierarchy, and Therapy Response in Pediatric Leukemia

BioRxiv, Mar 31, 2025

In our recent preprint, we investigate the role of the NUP98-NSD1 fusion oncogene in pediatric acute myeloid leukemia (AML), demonstrating that its expression in human hematopoietic stem and progenitor cells leads to leukemic transformation. We further elucidate that the oncogenic potential of NUP98-NSD1 is influenced by the ontogeny of the cells, highlighting the importance of developmental context in leukemia pathogenesis.

Models to study myelodysplastic syndrome and acute myeloid leukaemia

Current Opinion in Hematology, Nov 28, 2024

We recently wrote a review on the diverse models used to study myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML), highlighting their applications and limitations in understanding disease biology and testing therapies. The article focuses on genetically engineered mouse models, human iPSC-derived systems, patient-derived xenografts, and engineered blood stem cells, emphasizing their contributions to advancing translational hematology research.

Mapping the cellular origin and early evolution of leukemia in Down syndrome

Science, Jul 09, 2021

We utilized CRISPR/Cas9-mediated genome editing in human primary disomic and trisomic hematopoietic stem cells to generate an in vivo model of Down syndrome associated leukemia evolution. This humanized model enabled us to study the mechanism of predisposition, preleukemia initiation and leukemic transformation in order to identify potential therapeutic targets of the disease.

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