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 toxicity from high doses of chemotherapy and radiation therapy and development of a comprehensive late effects clinic for the long term follow up of transplant patients.
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
Associate Professor of Pediatrics (Stem Cell Transplantation)
Current Research and Scholarly Interests Genome Editing and Population Dynamics for Gene Therapy and Cancer Research
Maria Grazia Roncarolo
Professor of Pediatrics (Stem Cell Transplantation) and of Medicine (Blood and Marrow Transplantation)
Current Research and Scholarly Interests Research Interests
Immunetolerance: Mechanisms underlying T-cell tolerance, induction of T-cell anergy and regulatory T cells; Immunomodulation: mAbs, proteins and low molecular weight compounds which can modulate T-cell activation; Primary immunodeficiencies: Characterization of molecular and immunological defects; Gene therapy: Gene transduction of hematopoietic cells for gene therapy in primary immunodeficiencies and metabolic diseases; Hematopoiesis: Mechanisms underlying growth and differentiation of hematopoietic stem cells; Transplantation: Immune reconstitution and T-cell tolerance after allogenic stem cell transplantation; Cytokines/Cytokine receptors: Role in regulation of immune and inflammatory responses
Monogenic Autoimmune Disorders
Allogenic Bone Marrow Transplantation
Gene Therapy Clinical Trials
Cell Therapy Clinical Trials
Clinical Trials in Autoimmune Diseases and Organ Transplantation
Clinical Trials in Hemoglobinopathies
Instructor, Pediatrics - Stem Cell Transplantation
Bio I am currently postdoctoral research fellow pursuing immunotherapy research in the oncology department at Stanford University. My clinical training as a pediatric hematology oncology fellow at Memorial Sloan Kettering Cancer Center highlighted the desperate need for novel therapeutic options for a subtype of aggressive pediatric leukemia, Acute Myeloid Leukemia (AML). Despite our best standard of care for AML, long term survival rates range from 50-60% with an unacceptably high relapse rate of 40%. The urgent need for novel treatments inspired me to pursue a research project in adoptive immunotherapy, genetically modifying Tcells to express artificial T cell receptors, termed chimeric antigen receptors (CARs), that target AML specific antigens. In parallel to my clinical training, I constructed an AML specific CAR and demonstrated its ability to redirect T cell function mediating eradication of AML cells. As the field of CAR therapy rapidly advances, novel methods to optimize this therapeutic modality are imperative. To this end, supported by research demonstrating superior antitumor function of naïve derived effector T cells compared to central memory derived effector T cells, I am investigating whether preferential modification of naïve T cells to express CARs will generate a T cell subpopulation with increased efficacy. Consolidating my clinical and research experiences within highly academic institutes allows me to synthesize my pursuit of scientific rigor and commitment to the field of oncology, with a mission to achieve productive research and translatable results.