Institute for Stem Cell Biology and Regenerative Medicine
Showing 11-20 of 50 Results
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Asiri Ediriwickrema MD, PhD
Assistant Professor of Medicine (Hematology)
Current Research and Scholarly InterestsWe study hematopoiesis which is the complex process of producing blood cells that are essential for maintaining our basic health and well being. Our mission is to learn how these individual cells change as people age and develop cancer. Blood cell production is driven by the hematopoietic stem cell which gives rise to an incredible diversity of cells throughout life. Our research focuses on how dysregulation of this process leads to cytopenias and hematologic malignancies. We have expertise that spans clinical medicine, functional hematology, molecular and cellular biology, genomics, bioinformatics, and machine learning. By integrating advanced experimental and computational methods, we are examining blood cell development and function at single-cell resolution to advance patient diagnostics and treatment.
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Natalia Gomez-Ospina
Assistant Professor of Pediatrics (Genetics)
Current Research and Scholarly Interests1. Genome Editing and HSPC-Based Therapeutics
Dr. Gomez-Ospina’s research focuses on developing autologous hematopoietic stem and progenitor cell therapies using CRISPR/Cas9-mediated genome editing. Her work includes targeted integration of therapeutic transgenes into genomic safe-harbor loci, optimization of editing efficiency and safety, and development of transplantation and conditioning strategies that preserve long-term engraftment. A major goal is to create reusable, mutation-agnostic therapeutic platforms that can be adapted across diseases. Her translational work also extends to GMP manufacturing, analytical development, and IND-enabling studies needed to move genome-edited HSPC products toward clinical trials.
2. Neurometabolic and Neurodegenerative Disease Therapeutics
A major area of interest is the development of definitive therapies for lysosomal storage disorders and other neurogenetic diseases, including MPS I, Gaucher disease, Krabbe disease, progranulin deficiency, and Friedreich’s ataxia. Her laboratory investigates how hematopoietic-derived cells can deliver therapeutic proteins to peripheral tissues and the central nervous system. This includes strategies to enhance microglial replacement, improve CNS engraftment, and harness myeloid cells as therapeutic vehicles. The broader objective is to establish cell-based approaches capable of treating both inherited neurometabolic disorders and more common neurodegenerative diseases.
3. Rare Disease Genetics, Functional Genomics, and Lysosome Biology
Dr. Gomez-Ospina also studies the molecular basis of rare and undiagnosed genetic disorders. Through clinical genetics, international collaborations, and the NIH Undiagnosed Diseases Network, her work contributes to novel disease-gene discovery, functional validation of candidate variants, and definition of genotype–phenotype relationships. Her laboratory also investigates lysosomal biology and develops systems-level approaches, including the Lysosome Disease Atlas, to connect lysosome-associated genes with cellular function and human phenotypes. These efforts aim to improve diagnosis while identifying new disease mechanisms and therapeutic opportunities.
For more information go to our website:
https://www.gomezospina.com/ -
Stefan Heller, PhD, MS
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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Siddhartha Jaiswal
Associate 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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Calvin Kuo
Maureen Lyles D'Ambrogio Professor
Current Research and Scholarly InterestsWe study cancer biology, intestinal stem cells (ISC), and angiogenesis. We use primary organoid cultures of diverse tissues and tumor biopsies for immunotherapy modeling, oncogene functional screening and stem cell biology. Angiogenesis projects include blood-brain barrier regulation, stroke therapeutics and anti-angiogenic cancer therapy. ISC projects apply organoid culture and ko mice to injury-inducible vs homeostatic stem cells and symmetric division mechanisms.
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Kyle Loh
Associate Professor of Developmental Biology (Stem Cell)
BioHow the richly varied cell-types in the human body arise from one embryonic cell is a marvel and mystery. We have mapped how human embryonic stem cells develop into over twenty different types of cells. This roadmap allowed us to generate enriched populations of human brain, blood, blood vessel, bone, and other cells in a Petri dish from embryonic stem cells, with implications for developmental biology, stem cell biology, and regenerative medicine. Additionally, we have an emerging interest in exploring deadly biosafety level 4 viruses together with our collaborators.
Kyle attended the County College of Morris and Rutgers, and received his Ph.D. from Stanford (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 faculty member at Stanford. Kyle is a Packard Fellow, Pew Scholar, Human Frontier Science Program Young Investigator and Baxter Foundation Faculty Scholar. His research has been recognized by the NIH Director's Early Independence Award, ISSCR Early Career Impact Award, Forbes 30 Under 30, Harold Weintraub Graduate Award, Hertz Foundation Thesis Prize and A*STAR Investigatorship.
