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Director, CBMFC
The Children's Hospital of Philadelphia
Abramson Research Center
3615 Civic Center Blvd, Room 302D

Miscellaneous Information

Miscellaneous Information:

With extended training in Pediatric Hematology, Oncology and Stem Cell Transplantation, my clinical work is now focused on children with bone marrow failure. I strive to provide excellence in care for families and patients across a wide spectrum of inherited and acquired conditions that lead to loss of blood formation. As a Physician Scientist, both clinical and scientific goals of my work are therefore directed at improving our understanding and broadening our treatment options for patients with bone marrow failure.

My clinical interests focus on diagnosis and treatment of bone marrow failure and specifically on improving diagnostic and therapeutic capabilities. Advances in genomic technologies provide exciting opportunity to streamline the frequently extended diagnostic work up of children with bone marrow failure. Along with colleagues at other institutions, we are spearheading efforts to leverage molecular technologies toward the development of tests that improve diagnostic certainty and timeliness of genetic bone marrow failure. We are also developing clinical trials to enhance treatment and post treatment surveillance options.

My laboratory has longstanding expertise in Fanconi Anemia (FA), a rare inherited genetic condition with prominent hematologic complications. With a training background in transplantation and hematopoietic stem cell biology, I hope to improve our understanding of the progressive hematopoietic failure that occurs in patients with FA. In addition to preclinical vector development for stem cell gene therapy, our more recent studies aim to reveal the origins of bone marrow failure in FA before birth. Our studies in murine models of FA indicate that a significant portion of hematopoietic stem cells are lost prenatally and during infancy. We are developing strategies that will ameliorate those losses, with the long term goal of reversing the successive erosion of the stem cell pool in children. Additional projects in the lab are focused on stem cell regulation by trafficking of extracellular vesicles in the bone marrow microenvironment.