Comprehensive Bone Marrow Failure Center Research Overview



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Bone Marrow Failure is generally a sign of hematopoietic stem cell (HSC) dysfunction or loss from the bone marrow. The underlying cause in patients is often unknown, and marrow failure in young children especially can be genetic. This continues to impede development of new therapies and has fueled interest among physicians, scientists, and patients in deepening our understanding of marrow failure in children and adults.

The Comprehensive Bone Marrow Failure (BMF) Center at Children’s Hospital of Philadelphia brings together physicians and researchers to provide state-of-the-art diagnostics, clinical care, and investigational studies, as well as basic research focused on acquired and inherited BMF. The center is closely affiliated with colleagues caring for adult BMF patients at the Hospital of the University of Pennsylvania.

For more information about the research areas below, please visit the Kurre Lab.

  • Failure of the hematopoietic system with stem cell losses is a near uniform occurrence in Fanconi Anemia. The etiology remains unknown, and we are using murine models to understand the underlying defect
  • Hematopoietic development in bone marrow failure diseases is poorly understood and fetal deficits remain to be elucidated. As hematopoietic stem cells undergo maturation to an adult phenotype well after birth, therapeutic opportunities that focus on fetal characteristics may reveal additional therapeutic opportunities.
  • Fanconi Anemia is potentially curable by hematopoietic stem cell gene therapy. We are actively investigating new strategies to realize the full curative potential and translate this to the clinic.
  • Extracellular vesicles are constitutively released by cells. The transmission of protein an RNA cargo to other cells in both paracrine and endocrine manner has revealed potent regulatory capacity. We are beginning to study their role in hematopoietic stem cells (HSCs) regulation in several loss and gain of function models.

For more information about the research areas below, please visit the Olson Lab.

  • The role of germline genetic predisposition syndromes in the development of pediatric myelodysplastic syndrome and acute myeloid leukemia
  • Patterns of HLA allele expression and clonal evolution in acquired aplastic anemia, and how these findings may serve as biomarkers for disease response as well as clues to the underlying immune basis of this disease
  • Investigation of how intrinsic pathways governing hematopoietic stem cell niche function can be harnessed to improve the efficiency and durability of donor engraftment after hematopoietic stem cell transplantation, particularly in the context of bone marrow failure syndromes.
  • Clinical trials of novel conditioning and T cell depletion strategies to improve survival and decrease allo-immune complications for patients with non-malignant hematologic disorders undergoing hematopoietic stem cell transplantation.

The long-term goal of the Babushok Laboratory to improve the outcomes of patients with bone marrow failure disorders through bench-to-bedside research aimed at elucidating mechanisms of hematopoietic failure and transforming this knowledge into rational therapies.