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  • Markus Grompe
2020

AAV-Mediated CRISPR/Cas9 Gene Editing in Murine Phenylketonuria

Richards, D. Y., Winn, S. R., Dudley, S., Nygaard, S., Mighell, T. L., Grompe, M. & Harding, C. O., Jun 12 2020, In : Molecular Therapy - Methods and Clinical Development. 17, p. 234-245 12 p.

Research output: Contribution to journalArticle

Open Access
3 Scopus citations

In Vivo Lineage Tracing of Polyploid Hepatocytes Reveals Extensive Proliferation during Liver Regeneration

Matsumoto, T., Wakefield, L., Tarlow, B. D. & Grompe, M., Jan 2 2020, In : Cell Stem Cell. 26, 1, p. 34-47.e3

Research output: Contribution to journalArticle

8 Scopus citations
2019

Diabetes relief in mice by glucose-sensing insulin-secreting human α-cells

Furuyama, K., Chera, S., van Gurp, L., Oropeza, D., Ghila, L., Damond, N., Vethe, H., Paulo, J. A., Joosten, A. M., Berney, T., Bosco, D., Dorrell, C., Grompe, M., Ræder, H., Roep, B. O., Thorel, F. & Herrera, P. L., Mar 7 2019, In : Nature. 567, 7746, p. 43-48 6 p.

Research output: Contribution to journalArticle

47 Scopus citations

Endoplasmic Reticulum Stress-Induced Upregulation of STARD1 Promotes Acetaminophen-Induced Acute Liver Failure

Torres, S., Baulies, A., Insausti-Urkia, N., Alarcón-Vila, C., Fucho, R., Solsona-Vilarrasa, E., Núñez, S., Robles, D., Ribas, V., Wakefield, L., Grompe, M., Lucena, M. I., Andrade, R. J., Win, S., Aung, T. A., Kaplowitz, N., García-Ruiz, C. & Fernández-Checa, J. C., Aug 1 2019, In : Gastroenterology. 157, 2, p. 552-568 17 p.

Research output: Contribution to journalArticle

Open Access
8 Scopus citations

Insights From Liver-Humanized Mice on Cholesterol Lipoprotein Metabolism and LXR-Agonist Pharmacodynamics in Humans

Minniti, M. E., Pedrelli, M., Vedin, L. L., Delbès, A. S., Denis, R. G. P., Öörni, K., Sala, C., Pirazzini, C., Thiagarajan, D., Nurmi, H. J., Grompe, M., Mills, K., Garagnani, P., Ellis, E. C. S., Strom, S. C., Luquet, S. H., Wilson, E. M., Bial, J., Steffensen, K. R. & Parini, P., Jan 1 2019, (Accepted/In press) In : Hepatology.

Research output: Contribution to journalArticle

Open Access
2 Scopus citations

Loss of Oxidation Resistance 1, OXR1, Is Associated with an Autosomal-Recessive Neurological Disease with Cerebellar Atrophy and Lysosomal Dysfunction

Wang, J., Rousseau, J., Kim, E., Ehresmann, S., Cheng, Y. T., Duraine, L., Zuo, Z., Park, Y. J., Li-Kroeger, D., Bi, W., Wong, L. J., Rosenfeld, J., Gleeson, J., Faqeih, E., Alkuraya, F. S., Wierenga, K. J., Chen, J., Afenjar, A., Nava, C., Doummar, D. & 5 others, Keren, B., Juusola, J., Grompe, M., Bellen, H. J. & Campeau, P. M., Dec 5 2019, In : American Journal of Human Genetics. 105, 6, p. 1237-1253 17 p.

Research output: Contribution to journalArticle

1 Scopus citations

Using a barcoded AAV capsid library to select for clinically relevant gene therapy vectors

Pekrun, K., De Alencastro, G., Luo, Q. J., Liu, J., Kim, Y., Nygaard, S., Galivo, F., Zhang, F., Song, R., Tiffany, M. R., Xu, J., Hebrok, M., Grompe, M. & Kay, M. A., Nov 14 2019, In : JCI Insight. 4, 22, e131610.

Research output: Contribution to journalArticle

Open Access
5 Scopus citations
2018

Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity

Paulk, N. K., Pekrun, K., Zhu, E., Nygaard, S., Li, B., Xu, J., Chu, K., Leborgne, C., Dane, A. P., Haft, A., Zhang, Y., Zhang, F., Morton, C., Valentine, M. B., Davidoff, A. M., Nathwani, A. C., Mingozzi, F., Grompe, M., Alexander, I. E., Lisowski, L. & 1 others, Kay, M. A., Jan 3 2018, In : Molecular Therapy. 26, 1, p. 289-303 15 p.

Research output: Contribution to journalArticle

42 Scopus citations

Combination therapy with atorvastatin and celecoxib delays tumor formation in a Fanconi anemia mouse model

Zhang, Q-S., Deater, M., Phan, N., Marcogliese, A., Major, A., Guinan, E. C. & Grompe, M., Jan 1 2018, (Accepted/In press) In : Pediatric Blood and Cancer.

Research output: Contribution to journalArticle

1 Scopus citations

Comprehensive human cell-type methylation atlas reveals origins of circulating cell-free DNA in health and disease

Moss, J., Magenheim, J., Neiman, D., Zemmour, H., Loyfer, N., Korach, A., Samet, Y., Maoz, M., Druid, H., Arner, P., Fu, K. Y., Kiss, E., Spalding, K. L., Landesberg, G., Zick, A., Grinshpun, A., Shapiro, A. M. J., Grompe, M., Wittenberg, A. D., Glaser, B. & 3 others, Shemer, R., Kaplan, T. & Dor, Y., Dec 1 2018, In : Nature Communications. 9, 1, 5068.

Research output: Contribution to journalArticle

62 Scopus citations

Inflammatory Cytokine TNFα Promotes the Long-Term Expansion of Primary Hepatocytes in 3D Culture

Peng, W. C., Logan, C. Y., Fish, M., Anbarchian, T., Aguisanda, F., Álvarez-Varela, A., Wu, P., Jin, Y., Zhu, J., Li, B., Grompe, M., Wang, B. & Nusse, R., Nov 29 2018, In : Cell. 175, 6, p. 1607-1619.e15

Research output: Contribution to journalArticle

34 Scopus citations

Long-Term Correction of Diabetes in Mice by In Vivo Reprogramming of Pancreatic Ducts

Wang, Y., Dorrell, C., Naugler, W. E., Heskett, M., Spellman, P., Li, B., Galivo, F., Haft, A., Wakefield, L. & Grompe, M., May 2 2018, In : Molecular Therapy. 26, 5, p. 1327-1342 16 p.

Research output: Contribution to journalArticle

10 Scopus citations

Monitoring liver damage using hepatocyte-specific methylation markers in cell-free circulating DNA

Lehmann-Werman, R., Magenheim, J., Moss, J., Neiman, D., Abraham, O., Piyanzin, S., Zemmour, H., Fox, I., Dor, T., Grompe, M., Landesberg, G., Loza, B. L., Shaked, A., Olthoff, K., Glaser, B., Shemer, R. & Dor, Y., Jun 21 2018, In : JCI Insight. 3, 12

Research output: Contribution to journalArticle

Open Access
13 Scopus citations
2017

A Drug Screen using Human iPSC-Derived Hepatocyte-like Cells Reveals Cardiac Glycosides as a Potential Treatment for Hypercholesterolemia

Cayo, M. A., Mallanna, S. K., Di Furio, F., Jing, R., Tolliver, L. B., Bures, M., Urick, A., Noto, F. K., Pashos, E. E., Greseth, M. D., Czarnecki, M., Traktman, P., Yang, W., Morrisey, E. E., Grompe, M., Rader, D. J. & Duncan, S. A., Apr 6 2017, In : Cell Stem Cell. 20, 4, p. 478-489.e5

Research output: Contribution to journalArticle

41 Scopus citations

Adult Mouse Liver Contains Two Distinct Populations of Cholangiocytes

Li, B., Dorrell, C., Canaday, P. S., Pelz, C., Haft, A., Finegold, M. & Grompe, M., 2017, (Accepted/In press) In : Stem Cell Reports.

Research output: Contribution to journalArticle

25 Scopus citations

Chronic Phenotype Characterization of a Large-Animal Model of Hereditary Tyrosinemia Type 1

Elgilani, F., Mao, S. A., Glorioso, J. M., Yin, M., Iankov, I. D., Singh, A., Amiot, B., Rinaldo, P., Marler, R. J., Ehman, R. L., Grompe, M., Lillegard, J. B., Hickey, R. D. & Nyberg, S. L., Jan 1 2017, In : American Journal of Pathology. 187, 1, p. 33-41 9 p.

Research output: Contribution to journalArticle

7 Scopus citations

Diagnosis and treatment of tyrosinemia type I: a US and Canadian consensus group review and recommendations

Chinsky, J. M., Singh, R., Ficicioglu, C., van Karnebeek, C. D. M., Grompe, M., Mitchell, G., Waisbren, S. E., Gucsavas-Calikoglu, M., Wasserstein, M. P., Coakley, K. & Scott, C. R., Dec 1 2017, In : Genetics in medicine : official journal of the American College of Medical Genetics. 19, 12

Research output: Contribution to journalReview article

37 Scopus citations

Fah knockout animals as models for therapeutic liver repopulation

Grompe, M., 2017, Advances in Experimental Medicine and Biology. Springer New York LLC, Vol. 959. p. 215-230 16 p. (Advances in Experimental Medicine and Biology; vol. 959).

Research output: Chapter in Book/Report/Conference proceedingChapter

10 Scopus citations

Genome-wide genetic and epigenetic analyses of pancreatic acinar cell carcinomas reveal aberrations in genome stability

Jäkel, C., Bergmann, F., Toth, R., Assenov, Y., Van Der Duin, D., Strobel, O., Hank, T., Klöppel, G., Dorrell, C., Grompe, M., Moss, J., Dor, Y., Schirmacher, P., Plass, C., Popanda, O. & Schmezer, P., Dec 1 2017, In : Nature Communications. 8, 1, 1323.

Research output: Contribution to journalArticle

17 Scopus citations

Glycoprotein 2 is a specific cell surface marker of human pancreatic progenitors

Cogger, K. F., Sinha, A., Sarangi, F., McGaugh, E. C., Saunders, D., Dorrell, C., Mejia-Guerrero, S., Aghazadeh, Y., Rourke, J. L., Screaton, R. A., Grompe, M., Streeter, P. R., Powers, A. C., Brissova, M., Kislinger, T. & Nostro, M. C., Dec 1 2017, In : Nature communications. 8, 1, 331.

Research output: Contribution to journalArticle

35 Scopus citations

Reprogramming human gallbladder cells into insulin-producing β-like cells

Galivo, F., Benedetti, E., Wang, Y., Pelz, C., Schug, J., Kaestner, K. H. & Grompe, M., Aug 2017, In : PloS one. 12, 8, e0181812.

Research output: Contribution to journalArticle

10 Scopus citations

Successful engraftment of human hepatocytes in uPA- SCID and FRG® KO mice

Foquet, L., Wilson, E. M., Verhoye, L., Grompe, M., Leroux-Roels, G., Bial, J. & Meuleman, P., 2017, Methods in Molecular Biology. Humana Press Inc., Vol. 1506. p. 117-130 14 p. (Methods in Molecular Biology; vol. 1506).

Research output: Chapter in Book/Report/Conference proceedingChapter

11 Scopus citations
2016

Age-dependent pancreatic gene regulation reveals mechanisms governing human β cell function

Arda, H. E., Li, L., Tsai, J., Torre, E. A., Rosli, Y., Peiris, H., Spitale, R. C., Dai, C., Gu, X., Qu, K., Wang, P., Wang, J., Grompe, M., Scharfmann, R., Snyder, M. S., Bottino, R., Powers, A. C., Chang, H. Y. & Kim, S. K., May 10 2016, In : Cell Metabolism. 23, 5, p. 909-920 12 p.

Research output: Contribution to journalArticle

78 Scopus citations

A universal system to select gene-modified hepatocytes in vivo

Nygaard, S., Barzel, A., Haft, A., Major, A., Finegold, M., Kay, M. A. & Grompe, M., Jun 8 2016, In : Science Translational Medicine. 8, 342, 342ra79.

Research output: Contribution to journalArticle

16 Scopus citations

Curative ex vivo liver-directed gene therapy in a pig model of hereditary tyrosinemia type 1

Hickey, R. D., Mao, S. A., Glorioso, J., Elgilani, F., Amiot, B., Chen, H., Rinaldo, P., Marler, R., Jiang, H., DeGrado, T. R., Suksanpaisan, L., O'Connor, M. K., Freeman, B. L., Ibrahim, S. H., Peng, K. W., Harding, C., Ho, C. S., Grompe, M., Ikeda, Y., Lillegard, J. B. & 2 others, Russell, S. J. & Nyberg, S. L., Jul 27 2016, In : Science Translational Medicine. 8, 349, 349ra99.

Research output: Contribution to journalArticle

25 Scopus citations

Efficient generation of pancreatic β-like cells from the mouse gallbladder

Wang, Y., Galivo, F., Pelz, C., Haft, A., Lee, J., Kim, S. K. & Grompe, M., Nov 1 2016, In : Stem Cell Research. 17, 3, p. 587-596 10 p.

Research output: Contribution to journalArticle

7 Scopus citations

Human islets contain four distinct subtypes of β cells

Dorrell, C., Schug, J., Canaday, P. S., Russ, H. A., Tarlow, B. D., Grompe, M. T., Horton, T., Hebrok, M., Streeter, P., Kaestner, K. H. & Grompe, M., Jul 11 2016, In : Nature Communications. 7, 11756.

Research output: Contribution to journalArticle

138 Scopus citations

Identification of tissue-specific cell death using methylation patterns of circulating DNA

Lehmann-Werman, R., Neiman, D., Zemmour, H., Moss, J., Magenheim, J., Vaknin-Dembinsky, A., Rubertsson, S., Nellgård, B., Blennow, K., Zetterberg, H., Spalding, K., Haller, M. J., Wasserfall, C. H., Schatz, D. A., Greenbaum, C. J., Dorrell, C., Grompe, M., Zick, A., Hubert, A., Maoz, M. & 11 others, Fendrich, V., Bartsch, D. K., Golan, T., Sasson, S. A. B., Zamir, G., Razin, A., Cedara, H., Shapiroo, A. M. J., Glaser, B., Shemer, R. & Dor, Y., Mar 29 2016, In : Proceedings of the National Academy of Sciences of the United States of America. 113, 13, p. E1826-E1834

Research output: Contribution to journalArticle

197 Scopus citations

Metformin improves defective hematopoiesis and delays tumor formation in Fanconi anemia mice

Zhang, Q. S., Tang, W., Deater, M., Phan, N., Marcogliese, A. N., Li, H., Al-Dhalimy, M., Major, A., Olson, S., Monnat, R. J. & Grompe, M., Dec 15 2016, In : Blood. 128, 24, p. 2774-2784 11 p.

Research output: Contribution to journalArticle

26 Scopus citations

Silent Tyrosinemia Type I Without Elevated Tyrosine or Succinylacetone Associated with Liver Cirrhosis and Hepatocellular Carcinoma

Blackburn, P. R., Hickey, R. D., Nace, R. A., Giama, N. H., Kraft, D. L., Bordner, A. J., Chaiteerakij, R., McCormick, J. B., Radulovic, M., Graham, R. P., Torbenson, M. S., Tortorelli, S., Scott, C. R., Lindor, N. M., Milliner, D. S., Oglesbee, D., Al-Qabandi, W., Grompe, M., Gavrilov, D. K., El-Youssef, M. & 5 others, Clark, K. J., Atwal, P. S., Roberts, L. R., Klee, E. W. & Ekker, S. C., Oct 1 2016, In : Human mutation. 37, 10, p. 1097-1105 9 p.

Research output: Contribution to journalArticle

10 Scopus citations

Single-Cell Mass Cytometry Analysis of the Human Endocrine Pancreas

Wang, Y. J., Golson, M. L., Schug, J., Traum, D., Liu, C., Vivek, K., Dorrell, C., Naji, A., Powers, A. C., Chang, K. M., Grompe, M. & Kaestner, K. H., Oct 11 2016, In : Cell Metabolism. 24, 4, p. 616-626 11 p.

Research output: Contribution to journalArticle

64 Scopus citations

Stem Cells and Regeneration in the Digestive System: Keystone Meeting

Soffers, J. H. M., Hansen, D., Sinagoga, K. L., Li, B., Martin, M. G., Wells, J., Grompe, M. & Li, L., Sep 1 2016, In : Gastroenterology. 151, 3, p. e6-e9

Research output: Contribution to journalArticle

Stem cells versus plasticity in liver and pancreas regeneration

Kopp, J. L., Grompe, M. & Sander, M., Feb 25 2016, In : Nature Cell Biology. 18, 3, p. 238-245 8 p.

Research output: Contribution to journalArticle

74 Scopus citations

TGF-β inhibition rescues hematopoietic stem cell defects and bone marrow failure in Fanconi anemia

Zhang, H., Kozono, D. E., O'Connor, K. W., Vidal-Cardenas, S., Rousseau, A., Hamilton, A., Moreau, L., Gaudiano, E. F., Greenberger, J., Bagby, G., Soulier, J., Grompe, M., Parmar, K. & D'Andrea, A. D., May 5 2016, In : Cell Stem Cell. 18, 5, p. 668-681 14 p.

Research output: Contribution to journalArticle

54 Scopus citations
2015

Adeno-associated virus finds its disease

Russell, D. W. & Grompe, M., Sep 29 2015, In : Nature genetics. 47, 10, p. 1104-1105 2 p.

Research output: Contribution to journalShort survey

19 Scopus citations

Directed differentiation of cholangiocytes from human pluripotent stem cells

Ogawa, M., Ogawa, S., Bear, C. E., Ahmadi, S., Chin, S., Li, B., Grompe, M., Keller, G., Kamath, B. M. & Ghanekar, A., Aug 10 2015, In : Nature biotechnology. 33, 8, p. 853-861 9 p.

Research output: Contribution to journalArticle

114 Scopus citations

Fibroblast Growth Factor Signaling Controls Liver Size in Mice with Humanized Livers

Naugler, W. E., Tarlow, B. D., Fedorov, L. M., Taylor, M., Pelz, C., Li, B., Darnell, J. & Grompe, M., Sep 1 2015, In : Gastroenterology. 149, 3, p. 728e15-740.e15

Research output: Contribution to journalArticle

50 Scopus citations

Novel surface markers directed against adult human gallbladder

Galivo, F. H., Dorrell, C. S., Grompe, M., Zhong, Y. P., Streeter, P. & Grompe, M., Jul 1 2015, In : Stem Cell Research. 15, 1, p. 172-181 10 p.

Research output: Contribution to journalArticle

2 Scopus citations

Oxymetholone therapy of fanconi anemia suppresses osteopontin transcription and induces hematopoietic stem cell cycling

Zhang, Q. S., Benedetti, E., Deater, M., Schubert, K., Major, A., Pelz, C., Impey, S., Marquez-Loza, L., Rathbun, R. K., Kato, S., Bagby, G. C. & Grompe, M., Jan 13 2015, In : Stem Cell Reports. 4, 1, p. 90-102 13 p.

Research output: Contribution to journalArticle

14 Scopus citations

The Sirt1 activator SRT3025 expands hematopoietic stem and progenitor cells and improves hematopoiesis in Fanconi anemia mice

Zhang, Q. S., Deater, M., Schubert, K., Marquez-Loza, L., Pelz, C., Sinclair, D. A. & Grompe, M., Jul 1 2015, In : Stem Cell Research. 15, 1, p. 130-140 11 p.

Research output: Contribution to journalArticle

9 Scopus citations
2014

Adult Liver Stem Cells

Grompe, M., Jan 1 2014, Essentials of Stem Cell Biology: Third Edition. Elsevier Inc., p. 309-327 19 p.

Research output: Chapter in Book/Report/Conference proceedingChapter

Bipotential adult liver progenitors are derived from chronically injured mature hepatocytes

Tarlow, B. D., Pelz, C., Naugler, W. E., Wakefield, L., Wilson, E. M., Finegold, M. J. & Grompe, M., Jan 1 2014, In : Cell Stem Cell. 15, 5, p. 605-618 14 p.

Research output: Contribution to journalArticle

243 Scopus citations

Clonal tracing of Sox9+ liver progenitors in mouse oval cell injury

Tarlow, B. D., Finegold, M. J. & Grompe, M., Jul 2014, In : Hepatology. 60, 1, p. 278-289 12 p.

Research output: Contribution to journalArticle

140 Scopus citations

Erratum: Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype (Nature Biotechnology DOI: 10.1038/nbt.2884)

Yin, H., Xue, W., Chen, S., Bogorad, R. L., Benedetti, E., Grompe, M., Koteliansky, V., Sharp, P. A., Jacks, T. & Anderson, D. G., Sep 1 2014, In : Nature biotechnology. 32, 9, 1 p.

Research output: Contribution to journalComment/debate

1 Scopus citations

Evaluation of resveratrol and N-acetylcysteine for cancer chemoprevention in a Fanconi anemia murine model

Zhang, Q. S., Marquez-Loza, L., Sheehan, A. M., Watanabe-Smith, K., Eaton, L., Benedetti, E., Major, A., Schubert, K., Deater, M., Joseph, E. & Grompe, M., Apr 1 2014, In : Pediatric Blood and Cancer. 61, 4, p. 740-742 3 p.

Research output: Contribution to journalArticle

10 Scopus citations

Extensive double humanization of both liver and hematopoiesis in FRGN mice

Wilson, E. M., Bial, J., Bial, G., Jensen, B., Greiner, D. L., Brehm, M. A. & Grompe, M., Nov 1 2014, In : Stem Cell Research. Part A, p. 404-412 9 p.

Research output: Contribution to journalArticle

77 Scopus citations

Fumarylacetoacetate hydrolase deficient pigs are a novel large animal model of metabolic liver disease

Hickey, R. D., Mao, S. A., Glorioso, J., Lillegard, J. B., Fisher, J. E., Amiot, B., Rinaldo, P., Harding, C. O., Marler, R., Finegold, M. J., Grompe, M. & Nyberg, S. L., Jul 2014, In : Stem Cell Research. 13, 1, p. 144-153 10 p.

Research output: Contribution to journalArticle

31 Scopus citations

Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype

Yin, H., Xue, W., Chen, S., Bogorad, R. L., Benedetti, E., Grompe, M., Koteliansky, V., Sharp, P. A., Jacks, T. & Anderson, D. G., Jun 2014, In : Nature biotechnology. 32, 6, p. 551-553 3 p.

Research output: Contribution to journalArticle

569 Scopus citations

Liver stem cells, where art thou?

Grompe, M., Jan 1 2014, In : Cell Stem Cell. 15, 3, p. 257-258 2 p.

Research output: Contribution to journalShort survey

56 Scopus citations

Pharmacologic inhibition of L-tyrosine degradation ameliorates cerebral dopamine deficiency in murine phenylketonuria (PKU)

Harding, C. O., Winn, S. R., Gibson, K. M., Arning, E., Bottiglieri, T. & Grompe, M., Sep 1 2014, In : Journal of inherited metabolic disease. 37, 5, p. 735-743 9 p.

Research output: Contribution to journalArticle

24 Scopus citations