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Bertaina Laboratory

Hematopoietic Stem Cell Transplantation and Leukemia

The Bertaina Laboratory is focused on identifying innovative approaches of stem cell transplantation and molecular/genomic characterization of leukemia stem cells in myeloid pediatric hematologic malignancies.

In particular, our group aims to understand how γδ T-cells immune reconstitution (especially after αβ T-cell depleted haploidentical stem cell transplantation) impact on the outcome of children affected by either malignant and life-threatening non-malignant disorders. γδ T-cells combine conventional adaptive features with rapid, innate-like responses that place them in the initiation phase of immune reactions. In addition, they recognize tumor cells without recourse to the classical major histocompatibility complex (MHC) presentation. For this reason, they are the ideal cellular target for strategies of immunotherapy.

We also are interested in identifying new biomarkers of graft-versus-host disease (GvHD), a severe, sometime fatal immune-mediate post-transplant complication. Increasing our basic science understanding, will help us to develop novel target to expand treatment options for this disease.  To do so, we are studying small molecules of RNA (microRNA), their target proteins and, in parallel, cytokines secreted in the serum. Our laboratory is planning to use a variety of exciting new tools and methods to unlock future discoveries.

Moreover, we are investigating Juvenile Myelomonocytic Leukemia (JMML), a rare and aggressive leukemia of early childhood characterized by aberrant proliferation of myelomonocytic cells, progressive anemia, thrombocytopenia, hepatosplenomegaly and high fetal hemoglobin levels. The prognosis of JMML is poor and, so far, the only curative treatment is allogeneic hematopoietic stem cell transplantation. This highlights the urgent need to develop novel therapeutic approaches able to improve the current treatment results in JMML patients. To address this goal, the mechanisms of JMML leukemogenesis must be elucidated in order to be pharmacologically targeted.

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Alice Bertaina, MD, PhD

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