School of Medicine
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Maria Grazia Roncarolo
George D. Smith Professor of Stem Cell and Regenerative Medicine and Professor of Medicine (Blood and Marrow Transplantation and Cellular Therapy)
Current Research and Scholarly InterestsResearch 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
Current Research and Scholarly InterestsMy current research focuses on the influence of the microbiome on clinical outcomes in the pediatric oncology, hematology, and bone marrow transplant populations.
Ami J. Shah
Clinical Professor, Pediatrics - Stem Cell Transplantation
BioI joined Stanford University in 2015 as a Clinical Professor of Pediatrics in the Division of Hematology/ Oncology, Stem Cell Transplantation and Regenerative Medicine, having completed my training in Pediatric Hematology/ Oncology at Childrens Hospital Los Angeles. My areas of clinical expertise have been in the areas of transplantation for immune deficiencies and immune reconstitution post HSCT. I have been actively involved with the care and treatment of children with primary immune deficiencies and work with the Primary Immune Deficiencies Consortium (PIDTC). I am very interested in cellular therapies as a treatment modality for rare genetic diseases. I currently am the PI for several gene therapy trials at Stanford for various disorders including cerebral adrenoleukodystrophy (cALD), Sickle Cell Anemia, Thalassemia and Pyruvate Kinase Deficiency. My other main areas of research have been in studying the late effects of patients following stem cell transplantation, in specific the neurocognitive function post HSCT. I have been involved with several national committees addressing the late effects of HSCT within the ASBMT and COG.
In addition to my research work in stem cell transplantation, I have been actively involved with mentorship and graduate medical education. I am currently the Program Director for the Hematology/ Oncology Fellowship and serve as a mentor through the Pediatric Mentoring Group.
Professor of Medicine (Blood and Marrow Transplantation and Cellular Therapy) and of Pediatrics (Stem Cell Transplantation)
Current Research and Scholarly InterestsTransplantation of defined populations of allogeneic hematopoietic cells. Specifically, the way in which hematopoietic cell grafts alter antigen specific immune responses to allo-, auto- and viral antigens. The cellular and molecular basis of resistance to engraftment of allogeneic hematopoietic stem cells.