Institute for Stem Cell Biology and Regenerative Medicine
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Ash A. Alizadeh, MD/PhD
Moghadam Family Professor
Current Research and Scholarly InterestsMy research is focused on attaining a better understanding of the initiation, maintenance, and progression of tumors, and their response to current therapies toward improving future treatment strategies. In this effort, I employ tools from functional genomics, computational biology, molecular genetics, and mouse models.
Clinically, I specialize in the care of patients with lymphomas, working on translating our findings in prospective cancer clinical trials.
Lay Teng Ang
Instructor, Institute for Stem Cell Biology and Regenerative Medicine
BioAs a stem cell biologist, I aim to understand the mechanisms through which stem cells differentiate into progressively specialized cell types and to harness this knowledge to artificially generate pure populations of desired cell types from stem cells. My work over the past ten years has centered on pluripotent stem cells (PSCs, which include embryonic and pluripotent stem cells), which can generate any of the hundreds of diverse cell types in the body. However, it has been notoriously challenging to guide PSCs to differentiate into a pure population of a given cell type. Current differentiation strategies typically generate heterogeneous cell populations unsuitable for basic research or clinical applications. To address this challenge, I mapped the cascade of branching lineage choices through which PSCs differentiate into various endodermal and mesodermal cell types. I then developed effective methods to differentiate PSCs into specific lineages by providing the extracellular signal(s) that specify a given lineage while inhibiting the signals that induce the alternate fate(s), enabling the generation of highly-pure human heart and bone (Loh & Chen et al., 2016; Cell) and liver (Loh & Ang et al., 2014; Cell Stem Cell) from PSCs. My laboratory currently focuses on differentiating human PSCs into liver progenitors (Ang et al., 2018; Cell Reports) and blood vessel cells (Ang et al., 2022; Cell).
I earned my Ph.D. jointly from the University of Cambridge and A*STAR and was subsequently appointed as a Research Fellow and, later, a Senior Research Fellow at the Genome Institute of Singapore. I then moved my laboratory to Stanford University as a Siebel Investigator and Instructor at the Stanford Institute for Stem Cell Biology & Regenerative Medicine. My laboratory has been supported by the Siebel Investigatorship, California Institute for Regenerative Medicine, and other sources.
The Ernest and Amelia Gallo Professor, Professor of Urology, of Developmental Biology and, by courtesy, of Chemical and Systems Biology
Current Research and Scholarly InterestsFunction of Hedgehog proteins and other extracellular signals in morphogenesis (pattern formation), in injury repair and regeneration (pattern maintenance). We study how the distribution of such signals is regulated in tissues, how cells perceive and respond to distinct concentrations of signals, and how such signaling pathways arose in evolution. We also study the normal roles of such signals in stem-cell physiology and their abnormal roles in the formation and expansion of cancer stem cells.
Instructor, Institute for Stem Cell Biology and Regenerative Medicine
Current Research and Scholarly InterestsMy goal is to generate universally transplantable human organs in research animals.
Michael F. Clarke, M.D.
Karel H. and Avice N. Beekhuis Professor of Cancer Biology
Current Research and Scholarly InterestsDr. Clarke maintains a laboratory focused on two areas of research: i) the control of self-renewal of normal stem cells and diseases such as cancer and hereditary diseases; and ii) the identification and characterization of cancer stem cells. His laboratory is investigating how perturbations of stem cell regulatory machinery contributes to human disease. In particular, the laboratory is investigating epigenetic regulators of self renewal, the process by which stem cells regenerate themselves.
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.
Thiago Almeida Pereira
Instructor, Institute for Stem Cell Biology and Regenerative Medicine
Current Research and Scholarly InterestsMy research focus on fibrosis pathogenesis, identifying key pathways for therapeutic intervention and biomarker discovery. I'm currently investigating the Hedgehog pathway in liver and lung fibrotic diseases, such as schistosomiasis mansoni, alcoholic and non-alcoholic fatty liver disease, viral hepatitis B and C, idiopathic pulmonary fibrosis. I'm also investigating the role of tumor associated macrophages and cancer associated fibroblasts in liver and head and neck cancers.
Professor of Medicine (Pulmonary, Allergy and Critical Care Medicine)
Current Research and Scholarly InterestsBasic and translational research in lung stem cell biology, cancer, pulmonary fibrosis, COPD, and acute lung injury/ARDS. Upper airway stem cell CRISPR gene correction followed by autologous stem cell transplantation to treat Cystic fibrosis. Using lung organoids and precision cut lung slice cultures of mouse and human lungs to study molecular regulation of lung stem cells. Using transgenic mice to visualize Wnt protein transmission from niche cell to stem cell in vivo.
Maximilian Diehn, MD, PhD
Jack, Lulu, and Sam Willson Professor and Professor of Radiation Oncology (Radiation Therapy)
Current Research and Scholarly InterestsMy laboratory focuses on two main areas: 1) cancer stem cell biology and 2) novel biomarkers for identifying the presence of malignant cells (diagnostic), predicting outcome (prognostic), and predicting response to therapy (predictive). Areas of study include cancers of the lung, breast, and gastrointestinal system. Clinically I specialize in the treatment of lung cancer and applications of stereotactic ablative radiotherapy and perform both prospective and retrospective clinical studies.
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.
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Edward C. and Amy H. Sewall Professor in the School of Medicine and Professor of Otolaryngology - Head & Neck Surgery (OHNS)On Partial Leave from 08/01/2023 To 09/30/2023
Current Research and Scholarly InterestsOur research focuses on the inner ear, from its earliest manifestation as one of the cranial placodes until it has developed into a mature and functioning organ. We are interested in how the sensory epithelia of the inner ear that harbor the sensory hair cells develop, how the cells mature, and how these epithelia respond to toxic insults. The overarching goal of this research is to find ways to regenerate lost sensory hair cells in mammals.
Assistant Professor of Pathology
Current Research and Scholarly InterestsWe identified a common disorder of aging called clonal hematopoiesis of indeterminate potential (CHIP). CHIP occurs due to certain somatic mutations in blood stem cells and represents a precursor state for blood cancer, but is also associated with increased risk of cardiovascular disease and death. We hope to understand more about the biology and clinical implications of CHIP using human and model system studies.
Assistant Professor of Developmental Biology (Stem Cell)
BioHow the richly varied cell-types in the human body arise from one embryonic cell is a biological marvel and mystery. We have mapped how human embryonic stem cells develop into over twenty different human cell-types. This roadmap allowed us to generate enriched populations of human liver, bone, heart and blood vessel precursors in a Petri dish from embryonic stem cells. Each of these tissue precursors could regenerate their cognate tissue upon injection into respective mouse models, with relevance to regenerative medicine. In addition to our interests in developmental and stem cell biology, we also interested in discovering the entry receptors and target cells of deadly biosafety level 4 viruses, together with our collaborators.
Kyle attended the County College of Morris and Rutgers University, and received his Ph.D. from Stanford University (working with Irving Weissman), with fellowships from the Hertz Foundation, National Science Foundation and Davidson Institute for Talent Development. He then continued as a Siebel Investigator, and later, as an Assistant Professor and The Anthony DiGenova Endowed Faculty Scholar at Stanford, where he is jointly appointed in the Department of Developmental Biology and Institute for Stem Cell Biology & Regenerative Medicine. Kyle is a Packard Fellow, Pew Scholar, Human Frontier Science Program Young Investigator and Baxter Foundation Faculty Scholar, and his research has been recognized by the NIH Director's Early Independence Award, Forbes 30 Under 30, Harold Weintraub Graduate Award, Hertz Foundation Thesis Prize and A*STAR Investigatorship.
Dr. Michael T. Longaker
Deane P. and Louise Mitchell Professor in the School of Medicine and Professor, by courtesy, of Materials Science and Engineering
Current Research and Scholarly InterestsWe have six main areas of current interest: 1) Cranial Suture Developmental Biology, 2) Distraction Osteogenesis, 3) Fibroblast heterogeneity and fibrosis repair, 4) Scarless Fetal Wound Healing, 5) Skeletal Stem Cells, 6) Novel Gene and Stem Cell Therapeutic Approaches.
Ravi Majeti MD, PhD
Director, Stanford Institute for Stem Cell Biology and Regenerative Medicine, RZ Cao Professor and Professor of Medicine (Hematology)
Current Research and Scholarly InterestsThe Majeti lab focuses on the molecular/genomic characterization and therapeutic targeting of leukemia stem cells in human hematologic malignancies, particularly acute myeloid leukemia (AML). Our lab uses experimental hematology methods, stem cell assays, genome editing, and bioinformatics to define and investigate drivers of leukemia stem cell behavior. As part of these studies, we have led the development and application of robust xenotransplantation assays for human hematopoietic cells.
Professor of Neurology and, by courtesy, of Neurosurgery, of Pediatrics, of Pathology and of Psychiatry and Behavioral Sciences
Current Research and Scholarly InterestsThe Monje Lab studies the molecular and cellular mechanisms of postnatal neurodevelopment. This includes microenvironmental influences on neural precursor cell fate choice in normal neurodevelopment and in disease states.
Hiromitsu (Hiro) Nakauchi
Professor of Genetics (Stem Cell)On Partial Leave from 09/01/2023 To 12/31/2023
Current Research and Scholarly InterestsTranslation of discoveries in basic research into practical medical applications
Assistant Professor of Biomedical Data Science
Current Research and Scholarly InterestsOur group develops computational strategies to study the phenotypic diversity, differentiation hierarchies, and clinical significance of tumor cell subsets and their surrounding microenvironments. Key results are further explored experimentally, both in our lab and through collaboration, with the ultimate goal of translating promising findings into the clinic.
Virginia and Daniel K. Ludwig Professor of Cancer Research
Current Research and Scholarly InterestsOur laboratory studies Wnt signaling in development and disease. We found recently that Wnt proteins are unusual growth factors, because they are lipid-modified. We discovered that Wnt proteins promote the proliferation of stem cells of various origins. Current work is directed at understanding the function of the lipid on the Wnt, using Wnt proteins as factors the expand stem cells and on understanding Wnt signaling during repair and regeneration after tissue injury.
Lucy Erin O'Brien
Associate Professor of Molecular and Cellular Physiology
Current Research and Scholarly InterestsMany adult organs tune their functional capacity to variable levels of physiologic demand. Adaptive organ resizing breaks the allometry of the body plan that was established during development, suggesting that it occurs through different mechanisms. Emerging evidence points to stem cells as key players in these mechanisms. We use the Drosophila midgut, a stem-cell based organ analogous to the vertebrate small intestine, as a simple model to uncover the rules that govern adaptive remodeling.
Anthony Oro, MD, PhD
Eugene and Gloria Bauer Professor
Current Research and Scholarly InterestsOur lab uses the skin to answer questions about epithelial stem cell biology, differentiation and carcinogenesis using genomics, genetics, and cell biological techniques. We have studied how hedgehog signaling regulates regeneration and skin cancer, and how tumors evolve to develop resistance. We study the mechanisms of early human skin development using human embryonic stem cells. These fundamentals studies provide a greater understanding of epithelial biology and novel disease therapeutics.
Professor of Neurosurgery, Emeritus
Current Research and Scholarly InterestsMembers of the Palmer Lab study the biology of neural stem cells in brain development and in the adult. Our primary goal is to understand how genes and environment synergize in influencing stem cell behavior during development and how mild genetic or environmental risk factors for disease may synergize in their detrimental effects on brain development or in the risk of neuronal loss in age-related degenerative disease.
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.
Associate Professor of Biology
Current Research and Scholarly InterestsCardiovascular developmental biology
Maria Grazia Roncarolo
George D. Smith Professor of Stem Cell and Regenerative Medicine and Professor of Medicine (Blood and Marrow Transplantation and Cellular Therapy)On Leave from 03/01/2023 To 02/29/2024
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
Associate Professor (Research) of Obstetrics and Gynecology (Reproductive and Stem Cell Biology)
Current Research and Scholarly InterestsThe thread of Ariadne that connects germ cells, preimplatation development and pluripotent stem cells is the focus of my research, with a specific interest in human development. My long-term goals are: 1. Understanding the biology of germ cells and and their ability to sustain early preimplantation development; 2. Understanding the mechanisms that regulate very early cell fate decisions in human embryos; 3. Understanding the biology of derivation and maintenance of Pluripotent Stem Cells
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.
Avram Goldstein Professor in the School of Medicine, Professor of Neurosurgery and, by courtesy, of Neurology and of Psychiatry and Behavioral Sciences
Current Research and Scholarly InterestsInformation transfer at synapses mediates information processing in brain, and is impaired in many brain diseases. Thomas Südhof is interested in how synapses are formed, how presynaptic terminals release neurotransmitters at synapses, and how synapses become dysfunctional in diseases such as autism or Alzheimer's disease. To address these questions, Südhof's laboratory employs approaches ranging from biophysical studies to the electrophysiological and behavioral analyses of mutant mice.
Instructor, Institute for Stem Cell Biology and Regenerative Medicine
BioMy research interests are focused on understanding the molecular basis of early development and stem cells, as it is often aberrations in stem cells or signaling mechanisms between tissues that lead to diseased states, including tumor development and cancer progression. Knowledge of stem cells and development is also critical to develop appropriate cell-based therapies for various diseases or injuries. My prior and current works take advantage of both traditional techniques (gene targeting, lineage tracing) and state-of-the-art technologies (Single cell RNA sequencing, Chromatin Immunoprecipitation (ChIP-seq), Imaging Mass Cytometry) to elucidate fundamental molecular mechanisms underlying signaling in tissue biology.
Virginia & D.K. Ludwig Professor of Clinical Investigation in Cancer Research, Professor of Pathology, and of Developmental Biology
Current Research and Scholarly InterestsStem cell and cancer stem cell biology; development of T and B lymphocytes; cell-surface receptors for oncornaviruses in leukemia. Hematopoietic stem cells; Lymphocyte homing, lymphoma invasiveness and metastasis; order of events from hematopoietic stem cells [HSC] to AML leukemia stem cells and blood diseases, and parallels in other tissues; discovery of tumor and pathogenic cell 'don't eat me' and 'eat me' signals, and translation into therapeutics.
Assistant Professor of Pathology
Current Research and Scholarly InterestsFibrotic diseases kill more people than cancer in this country and worldwide. We believe that scar-forming cells called fibroblasts are at the core of the fibrotic response in parenchymal organ fibrosis in the lung, liver, skin, bone marrow and tumor stroma. At the cellular level we think of fibrosis as a step wise process which implicates inflammation and fibrosis. We seek to identify new effective immune therapy targets to treat fibrotic diseases.
Professor of Pathology and, by courtesy, of Chemical and Systems Biology
Current Research and Scholarly InterestsEpigenetic Reprogramming, Direct conversion of fibroblasts into neurons, Pluripotent Stem Cells, Neural Differentiation: implications in development and regenerative medicine
Lorry Lokey Professor and Professor of Developmental Biology
Current Research and Scholarly InterestsThe precise and robust regulation of gene expression is a cornerstone for complex biological life. Research in our laboratory is focused on understanding how regulatory information encoded by the genome is integrated with the transcriptional machinery and chromatin context to allow for emergence of form and function during human embryogenesis and evolution, and how perturbations in this process lead to disease.