Bio

Roberson Parkman received his MD degree from Yale University in 1965 and after completing his residencies at Yale and the Children’s Hospital Boston and a research associateship at the National Institutes of Health, he joined the faculty of the Harvard Medical School where he helped establish the bone marrow transplant programs at the Childrens Hospital and the Peter Bent Brigham Hospital.. In 1983 he moved to the Children’s Hospital Los Angles where he established the bone marrow transplantation and research immunology programs. In 2014 he joined the pediatric faculty of Stanford University.

His 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. His 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 he has clinically investigated the potential of hematopoietic stem cell transplantation to treat initially primary immune deficiency diseases and later the metabolic diseases, which lead to his involvement in the early gene transfer clinical trials.

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Laboratory website

Center for Definitive and Curative Medicine (CDCM)

Bio

• Rosa recived her MD from the University of Turin in 1987. She completed a fellowship in general pediatrics immunology at the University of Turin in 1991. Following the completion of her pediatrics residency, Rosa received training in molecular and cellular immunology in France (UNICET, Lyon) and the United States (DNAX Research Institute of Molecular and Cellular Biology, Palo Alto) There, she was first exposed to the importance of integrating in depth laboratory research with clinical observations to develop a translational research approach to science. She worked for fifteen years at the San Raffaele Scientific Institute in the Telethon Institute for Gene Therapy (HSR-TIGET), where she focused on dissecting the genetic and immunological basis of primary immunodeficiencies with autoimmune manifestations that might be treated by gene therapy.

Her research challenges the limits of "inexplicable" and "untreatable" diseases, and apply current scientific knowledge to understand the mechanisms of impaired cellular immune function underlying the clinical manifestations in order to develop curative treatments. She investigates the genetic and immunological basis of diseases with autoimmunity or immune dysregulation in children. she believes 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. She plans 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.

Bio

Dr. Bertaina was Head of the Stem Cell Transplant Unit in the Department of Hematology and Oncology at the Bambino Gesù Children’s Hospital in Rome (this institution currently has the largest number of children transplanted with hematopoietic progenitors/stem cells in Europe). She completed her MD degree at the University of Pavia in Italy, her fellowship in hematopoietic stem cell transplantation (HSCT) at the Bambino Gesù Children’s Hospital in Rome, and her PhD degree in Immunology and Biotechnology at Tor Vergata University in Rome.

Dr. Bertaina is a well known expert in the field of allogeneic HSCT in pediatric patients affected by hematological malignancies or nonmalignant disorders. In particular, she has largely contributed to set-up the novel approach of graft manipulation based on the physical elimination of alfa/beta T cells and B cells. Dr. Bertaina has an excellent both clinical and biological expertise, as documented by the several papers that she has published in the field of pediatric hematology and oncology. Moreover, she has developed a robust expertise in the knowledge of the different aspects of immunological reconstitution of children given an allograft of hematopoietic stem cell, paying peculiar attention to the innate immunity.

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Agnieszka Czechowicz received her MD and PhD degrees from Stanford University School of Medicine in 2010/2011; completing graduate research with Prof. Irv Weissman in Developmental Biology and was both an HHMI and Soros Fellow.  Subsequently she completed her pediatrics residency at Boston Children’s Hospital and was a clinical fellow in pediatric hematology/oncology at the Dana Farber Cancer Institute, while simultaneously pursing postdoctoral research with Prof. Derrick Rossi at Boston Children’s Hospital/Harvard Medical School/Harvard Stem Cell Institute and was an Amy Potter Fellow.  She recently returned back to Stanford University in 2017 to join the pediatric stem cell transplant faculty.

Agnieszka has long been interested in how hematopoietic stem cells interact with their microenvironment and the principles guiding hematopoietic stem cell engraftment in bone marrow transplantation.  She has done pioneering work highlighting that host hematopoietic stem cells limit donor stem cell engraftment and has developed several novel antibody-based conditioning regimens to overcome this challenge, the most advanced of which is being tested in clinical trials at Stanford in immunodeficiency patients undergoing stem cell transplants.  She is passionate about teaching and mentoring trainees at various levels, and also is a champion of bridging science and medicine through developing innovative new therapies.

Bio

Matthew Porteus MD, PhD is an Associate Professor in the Department of Pediatrics and Institute of Stem Cell Biology and Regenerative Medicine at Stanford. He graduated Magna Cum Laude from Harvard University with a degree in “History and Science” for which he wrote his undergraduate thesis on social dimensions of the recombinant DNA controversy in the early 1970’s. He then received his MD/PhD degrees from Stanford University. He completed his Pediatric residency training at Boston Children’s Hosptial at Harvard and then his specialty training in Pediatric Hematology/Oncology at Boston Children’s Hospital/Dana Farber Cancer Institute. He completed a post-doctoral research fellowship at the Caltech under the mentorship of Nobel Laureate Dr. David Baltimore.

His clinical interests on the Pediatric Stem Cell Transplant service are to develop improved methods of curing patients with genetic diseases using stem cell based therapies as well as reducing the complications from allogeneic stem cell transplants. His laboaratory research focus is on developing genome editing as an approach to cure disease, particularly those of the blood but also of other organ systems as well. His research program has made important discoveries in advancing the field of genome editing including the first use of genome editing using engineered nucleases in human cells and optimizing the use of the CRISPR/Cas9 system in primary human stem cells. His goal is to combine his research and clinical interests to bring innovative curative therapies to patients. He served on the National Academy Study Committee of Human Genome Editing.

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I did my training in Pediatrics and Hematology/ Oncology at Childrens Hospital Los Angeles where I was an attending physician for 15 years.. I joined Stanford University in 2015 as a clinical attending in the Division of Stem Cell Transplantation and Regenerative Medicine. My areas of clinical expertise have been in the areas of transplantation for immune deficiencies and immune reconstitution post HSCT. I was involved with several clinical gene therapy trial for SCID and ALD. My other main areas of research have been in the neurocognitive function post HSCT. I have been involved with several national committees addressing the late effects of HSCT within the ASBMT and COG.

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Katja Weinacht received her MD degree from Technische Universitaet Muenchen School of Medicine in 2002 and her PhD in medicine also from Technische Universitaet Muenchen in 2004. She was a postdoctoral fellow in the Channing Laboratory at Brigham and Women’s Hospital, Harvard Medical School and completaed her residency in pediatrics, at Massachusetts General Hospital, Harvard Medical School in 2009. She held a clinical pediatric fellowship in hematology/oncology at Boston Children’s Hospital, Dana-Farber Cancer Institute, Harvard Medical School from 2009-2012. Concurently, she held a research fellowship in pediatric immunology in the laboratory of Dr. Luigi D. Notarangelo at Boston Children’s Hospital, Harvard Medical School, from 2010-2012 where she acquired skills in the field of reprogramming, tissue engineering and gene correction. In 2016 she joined the pediatric faculty at Stanford University. 

Her special interest in the niche of diseases that intersect immune dysfunction and primary immunodeficiency. Her clinical practice focuses on pediatric patients requiring a hematopoietic stem cell transplantation, patients with DiGeorge Syndrome and patients with genetic immune diseases presenting with autoimmunity. As a physician-scientist, she strives to advance our insights into the mechanisms leading to immunodeficiency, autoimmunity and tolerance on a molecular level and to translates her research into novel targeted therapies patients.

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Dr. Weinberg recived his MD from Stanford University School of Medicine in 1978. Dr. Weinberg’s internship and residency in pediatrics was at the Montefiore Hospital and Medical Center from 1974-78, where he spent an additional year as Chief Resident. MHMC was a unique environment where daily advocacy for every child was instilled as an intrinsically worthy activity. During four monthly elective rotations at Memorial Sloan Kettering Cancer Center, he was exposed to children with cancer and felt called to a career in pediatric hematology-oncology. Dr. Weinberg’s fellowship was at Children’s Hospital Los Angeles from 1982-85. He entered the laboratories of Robertson Parkman and Peter Jones where he studied DNA methylation as a regulator of T-cell development by controlling expression of the T-cell receptor and CD3 complex. Dr. Parkman had just arrived at CHLA, and Dr. Weinberg was recruited as a faculty member in the newly formed Division of Research Immunology and Bone Marrow Transplantation.

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Dr. Shyr received his MD from Medical College of Wisconsin in 2005.  He completed his pediatric residency at UC San Diego in 2008 and his pediatric hematology/oncology at Children’s Hospital of Orange County in 2011.  Following his pediatric hem/onc fellowship, he received additional fellowship training in pediatric hematopoietic stem cell transplant at Texas Children’s Hospital/Baylor College of Medicine.  He has been a pediatric stem cell transplant attending physician at Johns Hopkin’s All Children’s Hospital (St. Petersburg, Florida) and Primary Children’s Hospital/University of Utah (Salt Lake City, Utah) before joining the pediatric stem cell transplant program at Stanford.  His clinical expertise and research interests focus on cellular therapy for non-malignant disorders including Wiskott-Aldrich Syndrome, severe combined Immunodeficiencies, leukodystrohies and Hurler’s Syndrome.   He is also developing research projects on applying machine learning methods to better predict transplant/cellular therapy related complications as well as post-transplant maintenance therapy for leukemias.

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I am a clinician and clinician scientist with an interest in translating potentially curative therapies to people with genetic diseases, particularly sickle cell disease and thalassemia. These therapies include standard and newer stem cell transplants as well as gene therapies. I performed the first gene therapy treatment for sickle cell disease at the Texas Medical Center in Houston in 2020 (via clinical trial) and am excited to translate more therapies at Stanford for sickle cell disease, thalassemia, and other rare genetic diseases. Additionally, I have an interest in understanding the long-term effects of stem cell therapies and participate as a member of STAR (Sickle Cell Transplant Advocacy and Research Alliance) to understand such. For all stem cell-based therapies, I strive to safely reduce toxicity related to the treatments whenever possible. Finally, I have a passion for global health and recognize disparities in health care access both within the US and abroad. Outside of North Carolina, Texas, and California, I have spent time learning and practicing medicine in Africa, India, and South and Central America witnessing the global effects of pediatric hematologic diseases. As a fierce advocate for members of the sickle cell community I strive to provide exceptional medical care considering the various complexities of this disease and safely expand access to disease modifying and corrective therapies to those interested and in need.