School of Medicine
Showing 1-10 of 23 Results
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 can be further applied to dissect underlying immunopathogenic mechanisms in many human disorders.
Currently, Dr. Cepika is a member of the laboratory of Dr. Maria Grazia Roncarolo, 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 mechanisms governing type 1 regulatory T (Tr1) cell differentiation and function, and use this knowledge to design Tr1 cell-based therapies for hematopoietic stem cell transplantation, cancer immunotherapy and autoimmunity.
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.
Assistant Professor of Pediatrics (Genetics) and of Pediatrics (Stem Cell Transplantation)
Current Research and Scholarly InterestsDr. Gomez-Ospina is a physician scientist and medical geneticist with a strong interest in the diagnosis and management of genetic diseases.
1) Lysosomal storage diseases:
Her research program is on developing better therapies for a large class of neurodegenerative diseases in children known as lysosomal storage disorders. Her current focus is on developing genome editing of hematopoietic stem cells as a therapeutic approach for these diseases beginning with Mucopolysaccharidosis type 1 and Gaucher disease. She established a genetic approach where therapeutic proteins can be targeted to a single well-characterized place in the genome known as a safe harbor. This approach constitutes a flexible, “one size fits all” approach that is independent of specific genes and mutations. This strategy, in which the hematopoietic system is commandeered to express and deliver therapeutic proteins to the brain can potentially change the current approaches to treating childhood neurodegenerative diseases and pave the way for alternative therapies for adult neurodegenerative disorders such as Alzheimer’s and Parkinson’s disease
2) Point of care ammonia testing
She also works in collaboration with other researchers at Stanford to develop point-of-care testing for serum ammonia levels. Such device will greatly improve the quality of life of children and families with metabolic disorders with hyperammonemia.
3) Gene discovery
Dr Gomez-Ospina lead a multi-institutional collaboration resulting in the discovery of a novel genetic cause of neonatal and infantile cholestatic liver disease. She collaborated in the description of two novel neurologic syndromes caused by mutations in DYRK1 and CHD4.
For more information go to our website:
Instructor, Pediatrics - Stem Cell Transplantation
BioI am a physician scientist at Stanford University/Lucile Packard Children’s Hospital, with a clinical and research focus in stem cell transplantation. Prior training includes earning MD and PhD degrees through the combined program at Saint Louis University School of Medicine, where I investigated the role of the cyclin dependent kinase inhibitor p21 in suppression of inflammatory cytokine production and treating inflammatory diseases. This project led to the publication of two first-author peer-reviewed articles, several middle-author publications, and a significant review article. I subsequently completed Pediatrics residency at the University of California Los Angeles/Mattel Children's Hospital and Pediatric Hematology/Oncology Fellowship at Stanford. Currently I'm an Instructor of Pediatrics in the division of Stem Cell Transplantation and Regenerative Medicine.
My long-term career goal is to develop a research program focusing on immune tolerance in stem cell transplantation and become a leader in the development of improved therapies for preventing or treating graft-versus-host disease. As such, I am excited about my ongoing work in the laboratory of Dr. Robert Negrin, focusing on the expansion and functional enhancement of regulatory T cells and invariant natural killer T cells. This work is currently supported by a St. Baldrick’s Foundation Fellowship, and has previously received funding from the Stanford Child Health Research Institute.
Associate Professor of Medicine (Blood and Marrow Transplantation and Cellular Therapy), of Pediatrics (Stem Cell Transplantation) and, by courtesy, of Surgery (Abdominal Transplantation)On Partial Leave from 01/01/2022 To 08/31/2022
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.