Markus Grompe, M.D.
- Professor of Pediatrics, School of Medicine
- Molecular and Medical Genetics Graduate Program, School of Medicine
- Cancer Biology Graduate Program, School of Medicine
- Program in Molecular and Cellular Biosciences, School of Medicine
Biography
Dr. Markus Grompe is the Ray Hickey Professor and Director of the Papé Family Pediatric Research Institute at Oregon Health & Science University in Portland, Oregon, USA. His research has focused on the use of in vivo selection to enhance gene and cell transplantation therapy for inherited diseases. Hepatic stem cells and their use in therapeutic liver repopulation are a main focus. To study the properties of human cells in a physiologic setting the laboratory has developed murine models supporting the expansion of human stem cells in transgenic mice. These humanized mice are an ideal platform for the development for novel therapeutics, including small molecules, gene therapy vectors and cells. He has published over 200 peer-reviewed articles and holds numerous patents.
Education and training
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Degrees
- M.D., 1983, University of Ulm Medical School
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Residency
- Pediatrics, Oregon Health & Science University, Portland, 1987
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Fellowship
- Genetics, Baylor College of Medicine, Houston, TX, 1991
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Certifications
- Clinical Genetics
Memberships and associations:
- Diplomat of the American Board of Human Genetics
Publications
Elsevier pure profilePublications
AAV capsid prioritization in normal and steatotic human livers maintained by machine perfusion
Nature biotechnologyComplete correction of murine phenylketonuria by selection-enhanced hepatocyte transplantation
HepatologyLong-term combination therapy with metformin and oxymetholone in a Fanconi anemia mouse model
Pediatric Blood and CancerA DNA methylation atlas of normal human cell types
NatureFanconi anemia-isogenic head and neck cancer cell line pairs
International Journal of CancerIn vivo tracing of the Cytokeratin 14 lineages using self-cleaving guide RNAs and CRISPR/Cas9
Developmental BiologyRapid in vivo multiplexed editing (RIME) of the adult mouse liver
HepatologyReplication stress increases mitochondrial metabolism and mitophagy in FANCD2 deficient fetal liver hematopoietic stem cells
Frontiers in OncologyStrong ubiquitous micro-promoters for recombinant adeno-associated viral vectors
Molecular Therapy Methods and Clinical DevelopmentThe DNA methylome of human vascular endothelium and its use in liquid biopsies
MedDevelopment of a Beta Cell-Specific Expression Control Element for Recombinant Adeno-Associated Virus
Human Gene TherapyHuman hepatocyte PNPLA3-148M exacerbates rapid non-alcoholic fatty liver disease development in chimeric mice
Cell ReportsMetformin for treatment of cytopenias in children and young adults with Fanconi anemia
Blood AdvancesSelf-cleaving guide RNAs enable pharmacological selection of precise gene editing events in vivo
Nature communicationsAAV integration in human hepatocytes
Molecular TherapyCancer stem cells
Annals of the New York Academy of SciencesDynamic Transcriptional and Epigenetic Changes Drive Cellular Plasticity in the Liver
HepatologyGeneration of functional ciliated cholangiocytes from human pluripotent stem cells
Nature communicationsInduced Liver Regeneration Enhances CRISPR/Cas9-Mediated Gene Repair in Tyrosinemia Type 1
Human Gene TherapyInhibition of TGFβ1 and TGFβ3 promotes hematopoiesis in Fanconi anemia
Experimental hematologyIn vitro expansion of cirrhosis derived liver epithelial cells with defined small molecules
Stem Cell ResearchLiver Injury Increases the Incidence of HCC following AAV Gene Therapy in Mice
Molecular TherapyMYC Promotes Bone Marrow Stem Cell Dysfunction in Fanconi Anemia
Cell Stem CellProliferative polyploid cells give rise to tumors via ploidy reduction
Nature communicationsTherapeutic liver repopulation by transient acetaminophen selection of gene-modified hepatocytes
Science translational medicineAAV-Mediated CRISPR/Cas9 Gene Editing in Murine Phenylketonuria
Molecular Therapy Methods and Clinical DevelopmentEfficient in vivo editing of OTC-deficient patient-derived primary human hepatocytes
JHEP ReportsInsights From Liver-Humanized Mice on Cholesterol Lipoprotein Metabolism and LXR-Agonist Pharmacodynamics in Humans
Hepatology