School of Medicine
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Associate Professor of Pediatrics (Stem Cell Transplantation) at the Lucile Salter Packard Children's Hospital
Current Research and Scholarly Interests Hematopoietic Stem cell biology-created a SCID mouse model to study engraftment of cord blood derived hematopoietic cells and use of this model to develop gene transfer technology for Fanconi anemia.
Clinical research interests are to develop new protocols to reduce graft vs host disease,treatment of viral infections post transplant and use of manipulated HSC graft in patients who receive mismatched donor transplants.
Professor of Pediatrics (Hematology/Oncology) at the Lucile Salter Packard Children's Hospital, Emeritus
Current Research and Scholarly Interests Bone marrow transplantation (BMT) is a treatment modality which is being broadly applied to a growing number of disorders. Increasing success with BMT is offering improved survival to pediatric and adult patients with acute leukemia, chronic leukemia, lymphomas, and a variety of solid tumors as well as severe aplastic anemia.
Associate Professor (Research) of Pediatrics (Stem Cell Transplantation)
Current Research and Scholarly Interests In the coming years, I plan to further determine the genetic and immunological basis of diseases with autoimmunity or immune dysregulation in children. I believe that much can still be learned from the in depth mechanistic studies of pediatric autoimmune diseases. Genomic analysis of the patients' samples has become possible which may provide a rapid indication of altered target molecules. I plan to implement robust functional studies to define the consequences of these genetic abnormalities and bridge them to the patient's clinical phenotype.
Understanding functional consequences of gene mutations in single case/family first and then validating the molecular and cellular defects in other patients with similar phenotypes, will anticipate and complement cellular and gene therapy strategies.
Clinical Associate Professor, Pediatrics - Stem Cell Transplantation
Current Research and Scholarly Interests 1. Developing novel bone marrow transplant regimens for patients with hemoglobinopathies
2. Cord blood transplantation
Adjunct Professor, Pediatrics - Stem Cell Transplantation
Bio My principal research interests have been the assessment of the immunological consequences of hematopoietic stem cell transplantation including both acute and chronic graft versus host disease and immune reconstitution and the use of hematopoietic stem cell transplantation to treat genetic diseases. My laboratory was the first to suggest that chronic graft versus host disease was an autoimmune disease directed at histocompatibility antigens shared by donors and recipients. The observation leaded to the assessment of the role of thymic dysfunction in the pathogenesis of chronic graft versus host disease. As a pediatric immunologist I have investigated the role of hematopoietic stem cell transplantation initially in the treatment of primary immune deficiency diseases and later the treatment of metabolic diseases, which lead to my involvement in the early gene transfer clinical trials.
Associate Professor of Pediatrics (Stem Cell Transplantation)
Current Research and Scholarly Interests Genome Editing and Population Dynamics for Gene Therapy and Cancer Research