Mark J. Osborn, PhD

Assistant Professor, Department of Pediatrics

Mark J. Osborn

Contact Info

Office Phone 612-626-2961

Fax 612-626-4074

Office Address:
Pediatric BMT
Masonic Cancer Research Building, MCRB 460E
425 East River Parkway
Minneapolis, MN 55455

Mailing Address:
Pediatrics BMT
MMC 366 Mayo
8366A (Campus Delivery Code)
420 Delaware St SE
Minneapolis, MN 55455

PhD, Microbiology Biology, Immunology & Cancer Biology, University of Minnesota. Minneapolis, MN


Dr. Osborn is an Assistant Professor in the Department of Pediatrics, Division of Blood and Marrow Transplantation. He is also a member of the Cancer Center. Dr. Osborn received his PhD degree from the University of Minnesota in 2009 in the laboratory of Dr. Bruce R. Blazar, MD.

Awards & Recognition

  • Innovator in Basic Science Award, Department of Pediatrics, University of Minnesota (2017, 2021)
  • Children’s Cancer Research Fund Butterfly Award, Medical category (2015)
  • Basic Science Paper of the Year Award, University of Minnesota (2014-2015)
  • Meet the Investigator profile, Journal of Investigative Dermatology (2014)
  • Best abstract award, Pediatrics Research, Education, and Scholarship Symposium (PRESS), University of Minnesota Medical School (2009)
  • American Heart Association Pre-doctoral Fellowship (2005-2007)

Professional Associations

  • Member, American and European Societies for Gene and Cellular Therapy


Research Summary/Interests

Dr. Osborn’s current research is focused on gene and cellular therapy for disorders treated by hematopoietic cell transplantation including: Fanconi anemia, epidermolysis bullosa, and Hurler syndrome. This involves utilizing a patient’s own cells for precision gene targeting and correction of their disease causing mutation. Genome editing nucleases and multiple terminally differentiated and stem cell populations are utilized towards optimizing ex vivo cellular therapies.

Gene Therapy

- Non-viral gene transfer for the treatment of inborn errors of metabolism.

Genome Editing
- Correction of disease specific mutations in a precise manner using homologous recombination.

Cellular Therapy
- Ex vivo correction of murine and human adult stem cells.


A Selection of Dr. Osborn’s Recent Publications

  • Minicircle DNA is superior to plasmid DNA in eliciting antigen-specific CD8+ T-cell responses. Dietz WM, Skinner NE, Hamilton SE, Jund MD, Heitfeld SM, Litterman AJ, Hwu P, Chen ZY, Salazar AM, Ohlfest JR, Blazar BR, Pennell CA, Osborn MJ. Mol Ther. 2013 Aug;21(8):1526-35. doi: 10.1038/mt.2013.85. Epub 2013 May 21.
  • TALEN-based gene correction for epidermolysis bullosa. Osborn MJ, Starker CG, McElroy AN, Webber BR, Riddle MJ, Xia L, DeFeo AP, Gabriel R, Schmidt M, von Kalle C, Carlson DF, Maeder ML, Joung JK, Wagner JE, Voytas DF, Blazar BR, Tolar J. Mol Ther. 2013 Jun;21(6):1151-9. doi: 10.1038/mt.2013.56. Epub 2013 Apr 2.
  • Keratinocytes from induced pluripotent stem cells in junctional epidermolysis bullosa. Tolar J, Xia L, Lees CJ, Riddle M, McElroy A, Keene DR, Lund TC, Osborn MJ, Marinkovich MP, Blazar BR, Wagner JE. J Invest Dermatol. 2013;133(2):592-5. Epub 2012 Aug 30.
  • Targeting G with TAL effectors: a comparison of activities of TALENs constructed with NN and NK repeat variable di-residues. Christian ML, Demorest ZL, Starker CG, Osborn MJ, Nyquist MD, Zhang Y, Carlson DF, Bradley P, Bogdanove AJ, Voytas DF. PLoS One. 2012;7(9):e45383. doi: 10.1371/journal.pone.0045383. Epub 2012 Sep 24.
  • Synthetic zinc finger nuclease design and rapid assembly. Osborn MJ, DeFeo AP, Blazar BR, Tolar J. Hum Gene Ther. 2011 Sep;22(9):1155-65. doi: 10.1089/hum.2011.072. Epub 2011 Aug 10.

Click here to see a complete list of Dr. Osborn’s studies which have been published by academic and research journals.