School of Medicine
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Professor of Pediatrics (Stem Cell Transplantation)
Current Research and Scholarly InterestsHematopoietic 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 InterestsBone 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 InterestsIn 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.
For further information please visit the Bacchetta Lab website:
Alice Bertaina MD, PhD
Associate Professor of Pediatrics (Stem Cell Transplantation)
Current Research and Scholarly InterestsDr. Bertaina is a highly experienced clinician and will play a key role in supporting Section Chief Dr. Rajni Agarwal and Clinical Staff in the Stem Cell Transplant Unit at Lucile Packard Children’s Hospital. She will also continue her research on immune recovery and miRNA, understanding the mechanisms underlying immune reconstitution, Graft-versus-Host Disease (GvHD), and leukemia relapse after allogeneic HSCT in pediatric patients affected by hematological malignant and non-malignant disorders.
Instructor, Pediatrics - Stem Cell Transplantation
BioDr. Cepika is an immunologist with an extensive background in translational research, autoimmunity, autoinflammation, and human systems immunology. Her goal is to understand the mechanisms governing immunological tolerance, and to leverage this knowledge to cure currently incurable diseases.
Dr. Cepika received her MD degree and a PhD in Immunology from the University of Zagreb School of Medicine in Croatia. There, she focused on the immunomonitoring of patients with lupus, identifying how circulating DNA levels changed with therapy. Subsequently, she joined the lab of Dr. Virginia Pascual at the Baylor Institute for Immunology Research in Dallas, Texas. Dr. Pascual had previously discovered that IL-1beta is a key pathogenic player in systemic juvenile idiopathic arthritis (sJIA), but the immune alterations contributing to IL-1beta-mediated inflammation remained unknown. To address this, Dr. Cepika developed a 3D in vitro stimulation assay to evaluate immune responses of blood leukocytes of pediatric sJIA patients. In combination with integrated bioinformatics analysis, this approach identified aberrant cellular responses, transcriptional pathways and genes that shed new light on immune dysregulation in sJIA. This assay (tollgene.org) can be further applied to dissect underlying immunopathogenic mechanisms in many human disorders.
Currently, Dr. Cepika is an Instructor in the Pediatric Division of Stem Cell Biology and Regenerative Medicine at Stanford University School of Medicine. There, she is working to uncover the underlying molecular mechanisms that govern the differentiation and function of antigen-inducible regulatory T cells called type 1 regulatory T (Tr1) cells, and use this knowledge to design Tr1 cell-based therapies to improve the outcomes of patients with cancer, autoimmunity, or receiving allogeneic cell or organ transplants.
Assistant Professor of Pediatrics (Stem Cell Transplantation)
Current Research and Scholarly InterestsDr. Czechowicz’s research is aimed at understanding how hematopoietic stem cells interact with their microenvironment in order to subsequently modulate these interactions to improve bone marrow transplantation and unlock biological secrets that further enable regenerative medicine broadly. This work can be applied across a variety of disease states ranging from rare genetic diseases, autoimmune diseases, solid organ transplantation, microbiome-augmentation and cancer.
Associate Professor of Medicine (Blood and Marrow Transplantation and Cellular Therapy), of Pediatrics (Stem Cell Transplantation) and, by courtesy, of Surgery (Abdominal Transplantation)
Current Research and Scholarly InterestsResearch focus in T cell immunotherapy and T cell immune monitoring using high-throughput sequencing and genomic approaches, with an emphasis on hematopoietic stem cell transplantation, the treatment of graft-versus-host disease and immune tolerance induction.
Adjunct Professor, Pediatrics - Stem Cell Transplantation
BioMy 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.