Photo of Markus Grompe, M.D.

Markus Grompe M.D.

  • (503) 494-8307
    • Professor of Pediatrics School of Medicine
    • Ray Hickey Chair of the of Pape' Family Pediatric Research Institute 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

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.


  • M.D., University of Ulm Medical School, 0 Germany 1983
  • Residency:

    • Pediatrics, Oregon Health & Science University, Portland, 1987
  • Fellowship:

    • Genetics, Baylor College of Medicine, Houston, TX, 1991
  • Certifications:

    • Clinical Genetics

Memberships and associations

  • Diplomat of the American Board of Human Genetics


  • "Long-Term Correction of Diabetes in Mice by In Vivo Reprogramming of Pancreatic Ducts." Molecular Therapy  In: , 01.01.2018.
  • "Combination therapy with atorvastatin and celecoxib delays tumor formation in a Fanconi anemia mouse model." Pediatric Blood and Cancer  In: , 01.01.2018.
  • "Glycoprotein 2 is a specific cell surface marker of human pancreatic progenitors." Nature Communications  In: , Vol. 8, No. 1, 331, 01.12.2017.
  • "Genome-wide genetic and epigenetic analyses of pancreatic acinar cell carcinomas reveal aberrations in genome stability." Nature Communications  In: , Vol. 8, No. 1, 1323, 01.12.2017.
  • "Diagnosis and treatment of tyrosinemia type I : a US and Canadian consensus group review and recommendations." Genetics in medicine : official journal of the American College of Medical Genetics  In: , Vol. 19, No. 12, 01.12.2017.
  • "A Drug Screen using Human iPSC-Derived Hepatocyte-like Cells Reveals Cardiac Glycosides as a Potential Treatment for Hypercholesterolemia." Cell Stem Cell  In: , Vol. 20, No. 4, 06.04.2017, p. 478-489.e5.
  • "Chronic Phenotype Characterization of a Large-Animal Model of Hereditary Tyrosinemia Type 1." American Journal of Pathology  In: , Vol. 187, No. 1, 01.01.2017, p. 33-41.
  • "Successful engraftment of human hepatocytes in uPA- SCID and FRG® KO mice."   Methods in Molecular Biology. Vol. 1506 Humana Press Inc., 2017. p. 117-130 (Methods in Molecular Biology; Vol. 1506).
  • "Adult Mouse Liver Contains Two Distinct Populations of Cholangiocytes." Stem Cell Reports  In: , 2017.
  • "Fah knockout animals as models for therapeutic liver repopulation."   Advances in Experimental Medicine and Biology. Vol. 959 Springer New York LLC, 2017. p. 215-230 (Advances in Experimental Medicine and Biology; Vol. 959).
  • "Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity." Molecular Therapy  In: , 2017.
  • "Reprogramming human gallbladder cells into insulin-producing β-like cells." PLoS One  In: , Vol. 12, No. 8, 2017, p. e0181812.
  • "Metformin improves defective hematopoiesis and delays tumor formation in Fanconi anemia mice." Blood  In: , Vol. 128, No. 24, 15.12.2016, p. 2774-2784.
  • "Efficient generation of pancreatic β-like cells from the mouse gallbladder." Stem Cell Research  In: , Vol. 17, No. 3, 01.11.2016, p. 587-596.
  • "Single-Cell Mass Cytometry Analysis of the Human Endocrine Pancreas." Cell Metabolism  In: , Vol. 24, No. 4, 11.10.2016, p. 616-626.
  • "Silent Tyrosinemia Type I Without Elevated Tyrosine or Succinylacetone Associated with Liver Cirrhosis and Hepatocellular Carcinoma." Human Mutation  In: , Vol. 37, No. 10, 01.10.2016, p. 1097-1105.
  • "Stem Cells and Regeneration in the Digestive System : Keystone Meeting." Gastroenterology  In: , Vol. 151, No. 3, 01.09.2016, p. e6-e9.
  • "Curative ex vivo liver-directed gene therapy in a pig model of hereditary tyrosinemia type 1." Science Translational Medicine  In: , Vol. 8, No. 349, 349ra99, 27.07.2016.
  • "Human islets contain four distinct subtypes of β cells." Nature Communications  In: , Vol. 7, 11756, 11.07.2016.
  • "A universal system to select gene-modified hepatocytes in vivo." Science Translational Medicine  In: , Vol. 8, No. 342, 342ra79, 08.06.2016.
  • "Age-dependent pancreatic gene regulation reveals mechanisms governing human β cell function." Cell Metabolism  In: , Vol. 23, No. 5, 10.05.2016, p. 909-920.
  • "TGF-β inhibition rescues hematopoietic stem cell defects and bone marrow failure in Fanconi anemia." Cell Stem Cell  In: , Vol. 18, No. 5, 05.05.2016, p. 668-681.
  • "Identification of tissue-specific cell death using methylation patterns of circulating DNA." Proceedings of the National Academy of Sciences of the United States of America  In: , Vol. 113, No. 13, 29.03.2016, p. E1826-E1834.
  • "Stem cells versus plasticity in liver and pancreas regeneration." Nature Cell Biology  In: , Vol. 18, No. 3, 25.02.2016, p. 238-245.
  • "Adeno-associated virus finds its disease." Nature Genetics  In: , Vol. 47, No. 10, 29.09.2015, p. 1104-1105.
  • "Fibroblast Growth Factor Signaling Controls Liver Size in Mice with Humanized Livers." Gastroenterology  In: , Vol. 149, No. 3, 01.09.2015, p. 728e15-740e15.
  • "Directed differentiation of cholangiocytes from human pluripotent stem cells." Nature Biotechnology  In: , Vol. 33, No. 8, 10.08.2015, p. 853-861.
  • "Novel surface markers directed against adult human gallbladder." Stem Cell Research  In: , Vol. 15, No. 1, 01.07.2015, p. 172-181.
  • "The Sirt1 activator SRT3025 expands hematopoietic stem and progenitor cells and improves hematopoiesis in Fanconi anemia mice." Stem Cell Research  In: , Vol. 15, No. 1, 01.07.2015, p. 130-140.
  • "Oxymetholone therapy of fanconi anemia suppresses osteopontin transcription and induces hematopoietic stem cell cycling." Stem Cell Reports  In: , Vol. 4, No. 1, 13.01.2015, p. 90-102.

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