Institute for Stem Cell Biology and Regenerative Medicine
Showing 11-20 of 36 Results
-
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/ -
Stefan Heller
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 09/30/2023Current 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.
-
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.
-
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.
-
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.
-
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.
-
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
-
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.
