School of Medicine
Showing 1-55 of 55 Results
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Rajni Agarwal
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 Toxicity from the conditioning regimens for stem cell transplants, reducing graft vs host disease, treatment of viral infections post transplant and use of manipulated HSC graft in patients who receive haplo donor transplants. -
Michael Amylon
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.
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Rosa Bacchetta
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:
http://med.stanford.edu/bacchettalab.html -
Alice Bertaina MD, PhD
Lorry I. Lokey Professor
On Leave from 03/01/2024 To 04/30/2024Current 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.
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Alma-Martina Cepika
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. -
Agnieszka Czechowicz, MD, PhD
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.
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Everett Meyer
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.
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Robertson Parkman
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.
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Matthew Porteus
Sutardja Chuk Professor of Definitive and Curative Medicine
BioDr. Porteus was raised in California and was a local graduate of Gunn High School before completing A.B. degree in “History and Science” at Harvard University where he graduated Magna Cum Laude and wrote an thesis entitled “Safe or Dangerous Chimeras: The recombinant DNA controversy as a conflict between differing socially constructed interpretations of recombinant DNA technology.” He then returned to the area and completed his combined MD, PhD at Stanford Medical School with his PhD focused on understanding the molecular basis of mammalian forebrain development with his PhD thesis entitled “Isolation and Characterization of TES-1/DLX-2: A Novel Homeobox Gene Expressed During Mammalian Forebrain Development.” After completion of his dual degree program, he was an intern and resident in Pediatrics at Boston Children’s Hospital and then completed his Pediatric Hematology/Oncology fellowship in the combined Boston Chidlren’s Hospital/Dana Farber Cancer Institute program. For his fellowship and post-doctoral research he worked with Dr. David Baltimore at MIT and CalTech where he began his studies in developing homologous recombination as a strategy to correct disease causing mutations in stem cells as definitive and curative therapy for children with genetic diseases of the blood, particularly sickle cell disease. Following his training with Dr. Baltimore, he took an independent faculty position at UT Southwestern in the Departments of Pediatrics and Biochemistry before again returning to Stanford in 2010 as an Associate Professor. During this time his work has been the first to demonstrate that gene correction could be achieved in human cells at frequencies that were high enough to potentially cure patients and is considered one of the pioneers and founders of the field of genome editing—a field that now encompasses thousands of labs and several new companies throughout the world. His research program continues to focus on developing genome editing by homologous recombination as curative therapy for children with genetic diseases but also has interests in the clonal dynamics of heterogeneous populations and the use of genome editing to better understand diseases that affect children including infant leukemias and genetic diseases that affect the muscle. Clinically, Dr. Porteus attends at the Lucille Packard Children’s Hospital where he takes care of pediatric patients undergoing hematopoietic stem cell transplantation.
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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
Clinical Interests
Primary Immunodeficiencies
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 -
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. -
Katja Gabriele Weinacht, MD, PhD
Assistant Professor of Pediatrics (Stem Cell Transplantation and Regenerative Medicine)
Current Research and Scholarly InterestsPediatric Hematopoietic Stem Cell Transplantation
DiGeorge Syndrome
Genetic Immune Diseases
Immune Dysregulation -
John Fraser Wright
Professor (Research) of Pediatrics (Stem Cell Transplantation)
On Partial Leave from 01/01/2024 To 04/30/2024BioJ Fraser Wright, PhD
Dr. Wright received his PhD in 1989 from the University of Toronto (Biochemistry) for studies
characterizing the interaction of complement with IgM, and completed post-doctoral studies at INSERM
/ CENG Grenoble, France in molecular immunology focused on antigen processing and presentation. He
was awarded a CRCS/ MRC Scholarship, gaining faculty appointment at the University of Toronto. In
1996 he joined industry as a Scientist at Pasteur Sanofi, contributing there to the development of
vaccines and cancer immunotherapies, and subsequently as Director of Development and Clinical
Manufacturing at Avigen, a gene therapy company that pioneered AAV-based investigational gene
therapies for hemophilia and Parkinson’s disease. In 2004 he returned to academia, establishing and
directing the Clinical Vector Facility at the Center for Cellular and Molecular Therapeutics at Children’s
Hospital of Philadelphia, and gaining faculty appointment at the University of Pennsylvania Perelman
School of Medicine as professor of Pathology and Laboratory Medicine. Dr. Wright has contributed to
several clinical development programs in gene therapy, including for Luxturna and Kymriah, the first
gene therapies for a genetic (RPE65 deficiency) and non-genetic (CAR-T immunotherapy) disease,
respectively, approved in the United States, and for the first gene therapy clinical trial that delivered an
AAV-vectorized monoclonal antibody to human subjects for HIV passive immunity. He is a Co-founder of
Spark Therapeutics, serving there and subsequently at Axovant as Chief Technology Officer. In 2019 Dr.
Wright joined Stanford University as Professor of Pediatrics at The Center for Definitive and Curative
Medicine (CDCM). His research program aims to address key immunological barriers to gene therapy
through innovative approaches to viral vector design and generation, and to develop vectorized
antibodies for serious human diseases.
