R. Scott McIvor, PhD

Professor, Department of Genetics, Cell Biology and Development

R. Scott McIvor

Contact Info


Office Phone 612-626-1497

Lab Phone 612-626-1435

Office Address:
Genetics, Cell Biology and Dev
5-102 MolCellBio
420 Washington Ave SE
Minneapolis, MN 55455

Mailing Address:
Genetics, Cell Biology/Dev
Room 6-160 JacH
321 Church St SE
Minneapolis, MN 55455

Lab Address:
5-226 MCB

PhD, University of Minnesota, 1982



Gene therapy


Research Summary/Interests

Gene therapy The general research interest of Dr. McIvor's laboratory is gene therapy for genetic diseases and cancer. Specific research activities include: (i) Use of recombinant retroviral and lentiviral vectors for gene transfer into hematopoietic stem cells in the treatment of cancer and inherited disorders such as immunodeficiency diseases and lysosomal storage diseases; (ii) Introduction of drug-resistance genes into normal hematopoietic cells to protect against the toxic side effects of cancer chemotherapy; (iii) Use of adeno-associated virus for treatment of storage diseases and as a vector for gene transfer into the central nervous system in the treatment of neurological disorders; (iv) Adaptation of a novel vertebrate transposon ("Sleeping Beauty") to viral and non-viral delivery systems for gene therapy.


For a comprehensive list of recent publications, refer to PubMed, a service provided by the National Library of Medicine.

  • Punwani, D., M. Kawahara, J. Yu, U. Sanford, S. Roy, K. Patel, D.A. Carbonaro, A.D. Karlen, S. Khan, K. Cornetta, M. Rothe, A. Schambach, D.B. Kohn, H.L. Malech, R.S. McIvor, J.M. Puck, M.J. Cowan. 2016. Lentivirus mediated correction of Artemis-deficient severe combined immunodeficiency. Human Gene Therapy, in press.
  • Podetz-Pedersen, K.M., E. R. Olson, N.V. Somia, S.J. Russelland R.S. McIvor. 2016. A broad range of dose optima achieve high-level, long-term gene expression after hydrodynamic delivery of Sleeping Beauty transposons using hyperactive SB100x transposase. Molecular Therapy Nucleic Acids 19: e279.
  • Filareto, A., F. Rinaldi, R.W. Arpke, R. Darabi, J.J. Belanto, E.A. Toso, A.Z. Miller, J.M. Ervasti, R.S. McIvor, M. Kyba and R.C.R. Perlingeiro.2015.Pax3-induced expansion enables the genetic correction of dystrophic satellite cells. Skeletal Muscle5: 36.
  • Hyland, K.A., E.R. Olson and R.S. McIvor.2015.Sleeping Beauty-mediated drug resistance gene transfer in human hematopoietic progenitor cells.Human Gene Therapy, 26: 657-663.
  • Multhaup, M.M., K.M. Podetz-Pedersen, A.D. Karlen, N.V. Somia, B.R Blazar, M.J. Cowan, and R.S. McIvor.2015.Role of transgene regulation in ex vivo lentiviral correction of Artemis deficiency.Human Gene Therapy 26: 232-243.
  • Podetz-Pedersen, K.M., V. Vezys, N.V. Somia, S.J. Russelland R.S. McIvor. 2014. Cellular immune response against firefly luciferase after Sleeping Beauty mediated gene transfer in vivo. Human Gene Therapy 25: 955-65.
  • Schuster, D.J., L.R. Belur, M.S. Riedl, S.A. Schnell, K.M. Podetz-Pedersen, K.F. Kitto, R.S. McIvor, L. Vulchanova and C.A. Fairbanks. 2014. Supraspinal gene transfer by intrathecal adeno-associated virus serotype 5. Front Neuroanat 2014 8:66.
  • Schuster, D.J., J.A. Dykstra, M.S. Riedl, K.F. Kitto, L. Belur, R.S. McIvor, R.P. Elde, C.A. Fairbanks, and L. Vulchanova. 2014. Biodistribution of adeno-associated virus serotype 9 (AAV9) after intrathecal and intravenous delivery in mouse. Front Neuroanat 2014 8:42.
  • Moriarity B.S., E.P. Rahrmann, D.A. Beckmann, C.B. Conboy, A.L. Watson, D.F. Carlson, E.R. Olson, K.A. Hyland, S.C. Fahrenkrug, R.S. McIvor, and D.A. Largaespada. 2014. Simple and efficient methods for enrichment and isolation of endonuclease modified cells. PLoS One 9:e96114.
  • Janson C.G., L.G. Romanova, P. Leone P, Z. Nan, L. Belur, R.S. McIvor, W. Low.2014. Comparison of Endovascular and Intraventricular Gene Therapy with AAV5-IDUA for Hurler Disease.Neurosurgery 74: 99-111.
  • Aronovich, E.L., B.C. Hall, J.B. Bell, R.S. McIvor, and P.B. Hackett. Quantitative Analysis of ?-L-Iduronidase Expression in Immunocompetent Mice Treated with the Sleeping Beauty Transposon System. PLoS One, in press.
  • Suksanpaisan, L., L. Pham, S. McIvor, S.J. Russell and K-W Peng. Oral contrast enhances the resolution of in-life NIS reporter gene imaging. Cancer Gene Therapy, in press.
  • Gori, J.L., B.C. Beard, N.P. Williams, C. Ironside, D. Swanson, R.S. McIvor, and H.P. Kiem. 2013. In vivo protection of activated Tyr22-dihydrofolate reductase gene-modified canine T lymphocytes from methotrexate. J Gene Med. 15: 233-241.
  • Adil, M., L. Belur, R.M. Levine, A.W. Tisdale, R.S. McIvor, and E. Kokkoli. PR_b functionalized stealth liposomes for targeted delivery to metastatic colon cancer. Biomaterials Science 1: 393-401.
  • Schuster, D.J., J.A. Dykstra, M.S. Riedl, K.F. Kitto, C.N. Honda, R.S. McIvor, C.A. Fairbanks, and L. Vulchanova. 2013. Visualization of spinal afferent innervation in the mouse colon by AAV8-mediated GFP expression. Neurogastroenterol Motil. 25; e89–e100.
  • Filareto, A., S. Parker, R. Darabi, L. Borges, T. Schaaf, T. Mayerhofer, J.S. Chamberlain, J.M. Ervasti, R.S. McIvor, M. Kyba and R.C.R. Perlingeiro. 2013. An ex vivo gene therapy approach to treat muscular dystrophy using iPS cells. Nature Communications 4: 1549.
  • Wolf, D.A., A.W. Lenander, Z. Nan, E.A. Braunlin, K.M. Podetz-Pedersen, C.B. Whitley,P. Gupta, W.C. Low, and R.S. McIvor. 2012. Increased longevity and metabolic correction following syngeneic bone marrow transplantation in a murine model of mucopolysaccharidosis type I. Bone Marrow Transplantation47: 1235-1240.
  • Wolf, D.A., L.R. Hanson, Z. Nan, W.C. Low, W.H. Frey II, and R.S. McIvor. 2012. Lysosomal enzyme can bypass the blood-brain barrier and reach the CNS following intranasal Administration. Molecular Genetics and Metabolism106: 131-134.
  • Hyland, K.A., E.R. Olson, K.J. Clark, E.L. Aronovich, P.B. Hackett, B.R. Blazar, J. Tolar, and R.S. McIvor. 2011. Sleeping Beauty-mediated correction of Fanconi anemia type C. Journal of Gene Medicine 13: 462-469.
  • Wilber, A, F.U. Montoya, L. Hammer, B.S. Moriarity, A.M. Geurts, D.A. Largaespada, C.M. Verfaillie, R.S. McIvor, and U. Lakshmipathy. 2011. Efficient non-viral integration and stable gene expression in multipotent adult progenitor cells. Stem Cells International, 2011:717069.
  • Multhaup, M.M, S. Gurram, K.M. Podetz-Pedersen, A.D. Karlen, D.L. Swanson, N.V. Somia, P.B. Hackett, M.J. Cowan, and R.S. McIvor. 2011. Characterization of the human Artemis promoter by heterologous gene expression in vitro and in vivo. DNA and Cell Biology 30: 751-761.
  • Wolf, D.A., A.W. Lenander, Z. Nan, L.R. Belur, C.B. Whitley, P. Gupta, W.C. Low, and R.S. McIvor. 2011. Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I. Neurobiol. Disease 43: 123-133.
  • Belur L,R., K.M.Podetz-Pedersen, B.S. Sorenson, A.H. Hsu, J.B. Parker, C.S. Carlson, D.A. Saltzman, S. Ramakrishnan, and R.S. McIvor. 2011. Inhibition of angiogenesis and suppression of colorectal cancer metastatic to the liver using the Sleeping Beauty Transposon System. Mol Cancer 10 (1) 14.