The Comprehensive Bone Marrow Failure (BMF) Center at The 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 colleagues caring for adult BMF patients at the Hospital of the University of Pennsylvania.

Research Topic: Hematopoietic Stem Cells, Bone Marrow Failure, Aplastic Anemia, Fanconi Anemia, Gene Therapy

Dr. Kurre’s laboratory has longstanding expertise in Fanconi Anemia (FA), a genetic condition with prominent hematologic complications. With training in transplantation and hematopoietic stem cell biology, he hopes to improve our understanding of the progressive hematopoietic failure in patients with BMF and FA, and broaden diagnostic approaches, and develop next generation therapies.

Research Topic: Fanconi Anemia, Hematopoietic Stem Cells, Gene Therapy, Extracellular Vesicles, Bone Marrow Failure

The goal of his laboratory research program is to improve HSCT outcomes by identifying methods to decrease the incidence of graft failure following HSCT, which is a particularly critical challenge in the setting of transplant for non-malignant conditions such as bone marrow failure. Methods are being examined to enhance donor hematopoietic stem cell (HSC) engraftment efficiency following HSCT through enhancement of the functions of specialized areas of the bone marrow microenvironment, known hematopoietic stem cell niches. Currently, therapeutic strategies are being tested that can be applied...

Research Topic: Bone Marrow Failure, Aplastic Anemia, clonal hematopoiesis, pediatric myelodysplastic syndrome, acute myeloid leukemia, AML/MDS predisposition, paroxysmal nocturnal hemoglobinuria, Immune evasion, autoimmunity

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. In our previous studies, we have established an analytical platform that sensitively and reliably identifies somatic changes in hematopoietic cells of patients with bone marrow failure using comparative whole exome sequencing (WES), single nucleotide polymorphism array (SNP-A) analysis, and deep targeted sequencing of the Major...

Research Topic: Bone Marrow Failure, Aplastic Anemia, clonal hematopoiesis, pediatric myelodysplastic syndrome, acute myeloid leukemia, AML/MDS predisposition, paroxysmal nocturnal hemoglobinuria, Immune evasion, autoimmunity