M
Markus Grompe
Researcher at Oregon Health & Science University
Publications - 323
Citations - 37404
Markus Grompe is an academic researcher from Oregon Health & Science University. The author has contributed to research in topics: Fanconi anemia & Stem cell. The author has an hindex of 88, co-authored 313 publications receiving 34220 citations. Previous affiliations of Markus Grompe include Northwestern University & University of North Carolina at Chapel Hill.
Papers
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Journal ArticleDOI
Focal neurometabolic alterations in mice deficient for succinate semialdehyde dehydrogenase
K. M. Gibson,D. S. M. Schor,Maneesh Gupta,W. S. Guerand,Henry Senephansiri,T. Burlingame,H. Bartels,Boris M. Hogema,Boris M. Hogema,Teodoro Bottiglieri,Wolfgang Froestl,O. C. Snead,Markus Grompe,Cornelis Jakobs +13 more
TL;DR: It is concluded that SS ADH‐deficient mice represent a valid metabolic model of human SSADH deficiency, manifesting focal neurometabolic abnormalities which could provide key insights into pathophysiologic mechanisms.
Journal ArticleDOI
Subtyping Analysis of Fanconi Anemia by Immunoblotting and Retroviral Gene Transfer
Michael Pulsipher,Gary M. Kupfer,Dieter Naf,Ahmed Suliman,Jeng Shin Lee,Petra M. Jakobs,Markus Grompe,Hans Joenje,Colin A. Sieff,Eva C. Guinan,Richard C. Mulligan,Alan D. D'Andrea +11 more
TL;DR: The combination of immunoblot analysis and retroviral-mediated phenotypic correction of FA cells allows a rapid method of FA subtyping.
Journal ArticleDOI
Using a barcoded AAV capsid library to select for clinically relevant gene therapy vectors
Katja Pekrun,Gustavo de Alencastro,Qing-Jun Luo,Jun Liu,Youngjin Kim,Sean Nygaard,Feorillo Galivo,Feijie Zhang,Ren Song,Matthew Tiffany,Jianpeng Xu,Matthias Hebrok,Markus Grompe,Mark A. Kay +13 more
TL;DR: The generation of a chimeric AAV capsid (AAV-KP1) that facilitates transduction of primary human islet cells and human embryonic stem cell-derived β cells with up to 10-fold higher efficiency compared with previously studied best-in-class AAV vectors is described.
Journal ArticleDOI
Curative ex vivo liver-directed gene therapy in a pig model of hereditary tyrosinemia type 1
Raymond D. Hickey,Shennen A. Mao,Jaime Glorioso,Faysal Elgilani,Bruce Amiot,Harvey S. Chen,Piero Rinaldo,Ronald J. Marler,Huailei Jiang,Timothy R. DeGrado,Lukkana Suksanpaisan,Michael K. O'Connor,Brittany L. Freeman,Samar H. Ibrahim,Kah Whye Peng,Cary O. Harding,Chak Sum Ho,Markus Grompe,Yasuhiro Ikeda,Joseph B. Lillegard,Stephen J. Russell,Scott L. Nyberg +21 more
TL;DR: This study demonstrates correction of disease in a pig model of metabolic liver disease by ex vivo gene therapy, and concludes that further exploration of ex vivo hepatocyte genetic correction is warranted for clinical use.
Journal ArticleDOI
Tissue Stem Cells: New Tools and Functional Diversity
TL;DR: How major advances in the ability to identify and monitor these cells, which include genetic lineage tracing, FACS purification, and robust in vitro clonogenic assays, have changed the authors' view of their roles in many organs are discussed.