MICaB Graduate Program
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Jakub Tolar, M.D., Ph.D.
Department of Pediatrics
Charles University, Czech Republic, M.D., 1990
University of Minnesota, Ph.D. 1996
Stem cell gene therapy
Dr. Tolar’s research focuses on finding new ways of treating children with lethal diseases—cancer, inborn errors of metabolism, and devastating genetic disorders—using stem cell transplantation. He is also looking for safer and more effective methods of repairing and using a patient’s own cells in diseases such as mucopolysaccharidosis type I (MPS1H, Hurler syndrome), Fanconi anemia, and epidermolysis bullosa. Additional research interests include: reducing the negative effects of stem cell transplantation (such as using mesenchymal stromal cells (MSCs) for graft-versus-host disease), creation and use of induced pluripotent stem cells (iPCS), gene therapy using multipotent adult progenitor cells (MAPCs), and using transposons and zinc finger nuclease technology to repair genes.
Selected Recent Publications:
- Osborn MJ, Webber BR, Knipping F, Lonetree CL, Tennis N, DeFeo AP, McElroy AN, Starker CG, Lee C, Gabriel R, Merkel S, Lund TC, Kelly-Spratt KS, Jensen MC, Voytas DF, von Kalle C, Schmidt M, Hippen KL, Miller JS, Scharenberg AM, Tolar J, Blazar BR. 2016. Evaluation of TCR gene editing achieved by TALENs, CRISPR/Cas9 and megaTAL nucleases. Mol Ther. 24(3):570-81.
- Modiano JF, Lindborg BA, McElmurry RT, Lewellen M, Forster CL, Zamora EA, Schaack J, Bellgrau D, O'Brien TD, Tolar J. 2015. Mesenchymal stromal cells inhibit murine syngeneic anti-tumor immune responses by attenuating inflammation and reorganizing the tumor microenvironment. Cancer Immunol Immunother. 64(11):1449-60
- Osborn MJ, Gabriel R, Webber BR, DeFeo AP, McElroy AN, Jarjour J, Starker CG, Wagner JE, Joung JK, Voytas DF, von Kalle C, Schmidt M, Blazar BR, Tolar J. 2015. Fanconi anemia gene editing by the CRISPR/Cas9 system. Hum Gene Ther. 26(2):114-26.
- Tolar J, Wagner JE. 2015. A biologic Velcro patch. N Engl J Med. Clinical Implications of Basic Research. 372(4):382-4.
- Tolar J, McGrath JA, Xia L, Riddle M, Lees CJ, Eide C, Keene DR, Liu L, Osborn MJ, Lund TC, Blazar BR, Wagner JE. 2014. Patient-specific naturally gene-reverted induced pluripotent stem cells in recessive dystrophic epidermolysis bullosa. J Invest Dermatol. 134(5):1246-54.
- Tolar J, Wagner JE.2013. Allogeneic blood and bone marrow cells for the treatment of severe epidermolysis bullosa: repair of the extracellular matrix. Lancet. 382(9899):1214-23.