Institute for Stem Cell Biology and Regenerative Medicine
Showing 1-100 of 135 Results
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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. -
Chiara Anselmi
Postdoctoral Scholar, Stem Cell Biology and Regenerative Medicine
BioMy expertise is in the areas of regeneration, evolution, the nervous system and cell biology. I use a marine colonial tunicate, Botryllus schlosseri, characterized by having robust regenerative capabilities and an assayable and frequent (weekly) CNS (Central nervous system) tissue regeneration and loss throughout adult life. I believe that comparative studies on a simple chordate can help us elucidate the principal mechanisms that are the foundation of regeneration and aging.
I use a multidisciplinary methodology that integrates advanced single cell RNAseq, imaging, multi-parameter flow cytometric isolation of cellular populations and transplantation assays to elucidate the cellular and genetic changes associated with neuronal degeneration process in young and old colonies. -
Philip Beachy
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.
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Joydeep Bhadury
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.
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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.
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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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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.
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Tushar Desai
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.
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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.
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Francisco Galdos
MD Student, expected graduation Spring 2024
Ph.D. Student in Stem Cell Biology and Regenerative Medicine, admitted Summer 2017
MSTP StudentBioMy journey to pursue the physician-scientist track stems from an early fascination with biology and my family’s eight-year struggle to save my younger brother’s life. My brother was born with a complex congenital heart defect known as Hypoplastic Left Heart Syndrome (HLHS). In 2003, my brother received a heart transplant, but despite this, he passed away in 2004. Growing up, I strived to find an explanation for my brother’s congenital heart defect and became interested in medicine at a young age. Looking to pursue this goal, I attended the Michael DeBakey High School for Health Professions, a top pre-health and science public school, and in 2011 I was accepted to Harvard University where I pursued a major in the department of Stem Cell and Regenerative Biology. During my college years I took classes in developmental and stem cell biology where I became interested in the use of these fields to understand congenital defects. I began my research career in Drs. Caroline and Geoffrey Burn’s lab studying the development of the great vessels of the heart in zebrafish, Throughout my time at Harvard, I was awarded numerous fellowships that supported my undergraduate research, including the prestigious Amgen Scholars Fellowship that supported summer research in Dr. Michael Longaker’s lab at Stanford University. I pursued my undergraduate honors thesis in Dr. Richard Lee’s laboratory where I identified the role of Apolipoprotein E as a factor necessary for maintaining mature beta cell gene expression. These experiences culminated in my decision to apply to the physician-scientist program at Stanford University where I am currently training to pursue a career in academic medicine with the ultimate goal of practicing as a pediatric cardiologist and a university professor with an active laboratory. Currently, I am a graduate student in Dr. Sean Wu’s laboratory where I study the development of ventricular development in the heart using both bioinformatic approaches such as scRNA-seq and human induced pluripotent stem cells to study the development of the left and right ventricles. Through my work, I aim to understand the mechanisms that give rise to single ventricle congenital heart defects with the hope of making a difference in the lives of children born with these diseases.
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Heather Gentner
Director of Finance and Administration (SoM), Stem Cell Bio Regenerative Med Institute
Current Role at StanfordI am the Director of Finance and Administration for the Institute for Stem Cell Biology and Regenerative Medicine (ISCBRM) in the School of Medicine. I oversee and carry out administrative and financial related functions for the department.
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Natalia Gomez-Ospina
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:
https://www.gomezospina.com/ -
Tal Gordon
Postdoctoral Scholar, Stem Cell Biology and Regenerative Medicine
BioI am a zoologist and molecular biologist interested in the molecular basis of regeneration. My research focuses on stem cells and regeneration in ascidians, a group of marine invertebrates that represent the closest living relatives of the vertebrates. One of the main questions that motivate my research is whether regeneration capabilities lost during evolution can, at least to some extent, be re-acquired. As regeneration is not universal in the animal kingdom, I hypothesize that comparing regeneration in species with distinct regenerative capacities will lead to the discovery of key components of regeneration.
During my postdoc I intend to use comparative genomics to identify conserved cellular and molecular mechanisms that underlie ascidians’ regeneration. -
Stefan Heller
Edward C. and Amy H. Sewall Professor in the School of Medicine and Professor of Otolaryngology - Head & Neck Surgery (OHNS)
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.
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Francie Igboabuchi
Life Science Rsch Prof 1, Stem Cell Bio Regenerative Med Institute
Current Role at StanfordLife Science Research Professional I
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Siddhartha Jaiswal
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.
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Daniel Dan Liu
MD Student, expected graduation Spring 2024
Ph.D. Student in Stem Cell Biology and Regenerative Medicine, admitted Autumn 2020
MSTP Student
Ph.D. Minor, Computer ScienceBioDaniel received his bachelor's in molecular biology from Princeton University in 2018. His undergraduate research, conducted under the mentorship of Dr. Yibin Kang, centered around cancer metastasis and cancer stem cell biology. He is currently an MD-PhD candidate in the lab of Dr. Irving Weissman, where he researches human neural stem cells and primary brain malignancies.
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Kyle Loh
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.
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Wan-Jin Lu
Basic Life Res Scientist, Stem Cell Bio Regenerative Med Institute
BioDr. Wan-Jin Lu is a Research Scientist in Dr. Phil Beachy's lab. Wan-Jin grew up in Taiwan, obtained her B.S. in Zoology at National Taiwan University and completed her PhD in Genetics and Development at UT Southwestern in the lab of Dr. John Abrams. Her Ph.D. research involved the identification of the evolutionary conserved function of the tumor suppressor gene p53 that ensures the quality control of germ cells. She then moved to the Bay Area, where she was a Damon Runyon Postdoctoral Fellow in the Institute of Stem Cell Biology and Regenerative Medicine in the Beachy lab. Her work currently focuses on understanding the function of Hedgehog signaling in taste receptor cell homeostasis and delineating the mechanisms of taste receptor regeneration after chemotherapy-induced loss.
Since 2017, she has been collaborating with Tabula Muris And Tabula Sapiens Consortium to investigate taste receptor stem cell renewal and regeneration in the Beachy lab. Her work has received funding support from California Institute of Regenerative Medicine (CIRM), Thomas and Stacey Siebel Foundation, and NIH (R21 and R01). -
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.
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Michelle Monje
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.
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Hiromitsu (Hiro) Nakauchi
Professor of Genetics (Stem Cell)
On Partial Leave from 09/01/2023 To 12/31/2023Current Research and Scholarly InterestsTranslation of discoveries in basic research into practical medical applications
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Yusuke Nakauchi
Instructor, Institute for Stem Cell Biology and Regenerative Medicine
Current Research and Scholarly InterestsFrom 2005 to 2010, my work as a clinical hematology fellow allowed me to experience first-hand how scientific advances that started in a laboratory can transform patients' lives. While many of my patients were cured of their disease with allogeneic hematopoietic stem cell transplantation, underscoring the importance of anti-tumor immunotherapy in eradicating leukemia, I witnessed face-to-face their suffering from the long-term consequence of graft-versus-host disease (GVHD). This experience was ultimately what drove me to engage in research to discover novel therapies. For this reason, I embarked on a Ph.D. program in 2010 to design antibody therapy to (i) target GVHD and (ii) target hematological malignancies. Under the mentorship of Professor Hiromitsu Nakauchi at the University of Tokyo, an international leader in hematopoiesis, I developed allele-specific anti-human leukocyte antigen (HLA) monoclonal antibodies for severe GVHD caused by HLA-mismatched hematopoietic stem cell transplantation (Nakauchi et al., Exp Hematol, 2015). This study was the first to find that anti-HLA antibodies can be used therapeutically against GVHD. That success gave me the motivation and confidence to further my research beyond targeting GVHD to targeting leukemic stem cells through my postdoctoral fellowship in the laboratory of Professor Ravindra Majeti here at Stanford University.
Many people suffer from leukemia each year, but we still don't know how to cure it completely. Recent advances in sequencing technologies have tremendously improved our understanding of the underlying mutations that drive hematologic malignancies. However, the reality is that most of the mutations are not easily "druggable," and the discovery of these mutations has not yet significantly impacted patient outcomes. This is perhaps the most crucial challenge facing a translational cancer researcher like myself. My current research is a major step toward my long-term goal of making personalized medicine a reality for patients with acute myeloid leukemia (AML) and other hematologic malignancies.
Since joining the Majeti lab, I have been targeting the ten-eleven translocation methylcytosine dioxygenase-2 (TET2) mutation, which is aberrant in leukemia at a high rate and has been studied using human-derived cells. TET2 is known to be involved in the clonal expansion of cells, and people with this mutation are more likely to suffer from hematologic malignancies. It is also known to be involved in the development of coronary artery disease, a gene that has attracted much attention in recent studies. In my field, it is an essential gene involved in the abnormal proliferation of hematopoietic stem cells. Focusing on this gene, I mapped TET2-dependent 5hmC, epigenetic and transcriptional programs matched to competitive advantage, myeloid skewing, and reduced erythroid output in TET2-deficient hematopoietic stem and progenitor cells (HSPC). Vitamin C and azacitidine restore the 5hmC landscape and phenotypes in TET2-mutant HSPCs. These findings offer a comprehensive resource for TET-dependent transcriptional regulation of human hematopoiesis and shed light on the potential mechanisms by which TET deficiency contributes to clonal hematopoiesis and malignancies. Of course, these findings would also be of value in understanding the biology of normal hematopoietic stem cells (HSCs) and various other TET2-related cancers.
And from now on, I would like to use the single-cell transplantation techniques mastered in the Majeti lab to study the behavior of normal and aberrant human HSCs using various new methods, ultimately preventing the progression of AML.
In my clinical experience, I have lost many AML patients. With the regret and sadness of losing these patients in my heart, I hope to one day contribute to developing treatments that will fundamentally change how the world treats leukemia. -
Gernot Neumayer
Sr Res Scientist-Basic Life, Stem Cell Bio Regenerative Med Institute
BioI am a passionate scientist with expertise in basic and translational research related to ageing, cancer, genomic instability, DNA damage response (HDR & NHEJ), genome editing (CRISPR), regenerative medicine (iPSCs), cellular identity (reprogramming), and proteomics (interactions, biomarkers, target identification). My extensive experience is reflected by 10 peer reviewed publications. I possess excellent communication and technical writing skills (English/German), as evidenced by collaborations with world renowned institutions and >$460,000 won from scholarships, grants & awards. Recent highlights: Postdoctoral Young Investigator Award from Stanford University for scientific merit, commitment & leadership; “Played a big part” in securing a CRISPR-based $5.1Mio grant for regenerative medicine at Stanford University; Poster prize (out of 77 entries) at the Department of Pathology, Stanford University 2019 research day.
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Aaron Newman
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.
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Roeland Nusse
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.
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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.
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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.
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Theo Palmer
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.
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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.
