R. Scott McIvor, Ph.D. Professor Department of Genetics, Cell Biology and Development University of Minnesota, 1982, Ph.D. mcivo001@tc.umn.edu 612-626-1497 office 612-626-1435 lab

Research 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.

Selected Recent Publications:

• Score, P.R., L. Belur, J.L. Frandsen, J.L. Geurts, T. Yamaguchi, N.V. Somia, P.B. Hackett, D.A. Largaespada, and R.S. McIvor.   Sleeping Beauty-mediated transposition and long-term expression in vivo: application of the LoxP-Cre recombinase system for transposon-specific expression.  Molecular Therapy, in press.

• Wilber, A.C., J.L. Frandsen, J.L. Geurts, D.A. Largaespada, P.B. Hackett, and R.S. McIvor.  RNA as a source of transposase for Sleeping Beauty-mediated gene insertion and expression in somatic cells and tissues.  Molecular Therapy, in press.

• Belur, L., R.I. James, C. May, M.D. Diers, D. Swanson, R. Gunther and R.S. McIvor.  2005.   Methotrexate preconditioning allows sufficient engraftment to confer drug resistance in mice transplanted with marrow expressing drug-resistant dihydrofolate reductase activity.  Journal of Pharmacology and Experimental Therapeutics 314: 668-674.

• Hartung, S.D., J.S. Frandsen, D. Pan, B. Koniar, P. Graupman, R. Gunther, W.C. Low, C.B. Whitley and R. S. McIvor.  2004.  Correction of metabolic, neurologic, and craniofacial abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human a-L-iduronidase gene.  Molecular Therapy, 9: 866-875.

• Belur, L., J.L. Frandsen, A. Dupuy, D.H. Ingbar, D.L. Largaespada, P.B. Hackett and R.S. McIvor.  2003.  Integration and long-term expression in lung mediated by the Sleeping Beauty transposon system. Molecular Therapy 8: 501-507.

• Sweeney, C.L., J.L. Frandsen, C.M. Verfaillie and R.S. McIvor.  2003.  Trimetrexate inhibits progression of the murine 32Dp210 model of chronic myeloid leukemia in animals expressing drug-resistant dihydrofolate reductase.  Cancer Research 63:1304-10.

Last modified on: October 11, 2005