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:

• Vink C.A., H.B. Gaspar, R. Gabriel, M. Schmidt, R.S. McIvor, A.J. Thrasher, and W. Qasim. 2009. Sleeping Beauty Transposition From Nonintegrating Lentivirus. Molecular Therapy, 17: 1197-1204.
• Xue X., X. Huang X., S.E. Nodland, L. Mates, L. Ma, Z. Izsvak, Z. Ivics, T.W. Lebien, R.S. McIvor, J.E. Wagner, and X. Zhou. 2009. Stable gene transfer and expression in cord blood-derived CD34+ hematopoietic stem and progenitor cells by a hyperactive Sleeping Beauty transposon system. Blood, in press.
• Aronovich E.L., J.B. Bell, S.A. Khan, L.R. Belur, R. Gunther, B. Koniar, P.A. Schachern, J.B. Parker, C.S. Carlson, C.B. Whitley, R.S. McIvor, P. Gupta, and P.B. Hackett. 2009. Systemic Correction of Storage Disease in MPS I NOD/SCID Mice Using the Sleeping Beauty Transposon System. Molecular Therapy 17: 1136-1144
• Belur, L.R., Kaemmerer, W.F., McIvor, R.S., Low W.C. 2008. Adeno-associated virus type 2 vectors: transduction and long-term expression in cerebellar Purkinje cells in vivo is mediated by the fibroblast growth factor receptor 1. Archiv. Virol. 153: 2107-2110.
• Wangensteen, K.J., A. Wilber, V.W. Keng, Z. He, I. Matis, L. Wangensteen, C.M. Carlson, Y. Chen, C.J. Steer, R.S. McIvor, D.A. Largaespada, X.Wang, and S.C. Ekker. 2008. A facile method for somatic, lifelong manipulation of multiple genes in the mouse liver. Hepatology 47: 1714-1724.
• Gao, Y., T. Zu, W.C. Low, H.T. Orr, and R.S. McIvor. 2008. Antisense RNA sequences modulating the expression of ataxin-1 message: a molecular model of gene therapy for spinocerebellar ataxia type 1, a dominant-acting unstable trinucleotide repeat disease. Cell Transplantation, 17: 723-734
• Huang, X., H. Guo, J. Kang, S. Choi, T.C. Zhou, S. Tammana, C.J. Lees, Z.Z. Li, M. Milone, B.L. Levine, J. Tolar, C.H. June, R.S. McIvor, J.E. Wagner, B.R. Blazar, and X. Zhou. 2008. Sleeping Beauty transposon-mediated engineering of human primary T cells for therapy of CD19+ lymphoid malignancies. Molecular Therapy 16: 580-589.
• Wilber, A., J.L. Linehan, X. Tian, P.S. Woll, J. Morris, L.R. Belur, R.S. McIvor, and D.S. Kaufman. 2007. Efficient and stable transgene expression in human embryonic stem cells using transposon-mediated gene transfer. Stem Cells 25: 2919-2927
• Gori, J.L., K. Podetz-Pederson, D. Swanson, A.D. Karlen, R. Gunther N.V. Somia, and R.S McIvor. 2007. Protection of mice from methotrexate toxicity by ex vivo transduction using lentivirus vectors expressing drug-resistant dihydrofolate reductase. J. Pharmacol. & Exptl. Therapeutics 322: 989-997.
• Aronovich, E.L., J.B. Bell, L.R. Belur, R. Gunther, B. Koniar, D.C.C. Erickson, P.A. Schachern, I. Matise, R.S. McIvor, C.B. Whitley and P.B. Hackett. 2007. Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidoses. J. Gene Medicine 9: 403-415.
• Wilber, A., K.J. Wangensteen, Y. Chen, L. Zhuo, J.L. Frandsen, J. Bell, Z.J. Chen, S.C. Ekker, R.S. McIvor, and X. Wang. 2007. Messenger RNA as a Source of Transposase for Sleeping Beauty Transposon-mediated Correction of Hereditary Tyrosinemia Type I. Molecular Therapy 15: 1280-1287.
• Baliunas, D., K.J. Wangensteen, A. Wilber, J. Bell, A. Geurts, S. Sivasubbu, X. Wang, P.B. Hackett, D.A. Largaespada, R.S. McIvor, and S.C. Ekker. 2006 Harnessing an efficient large cargo-capacity transposon for vertebrate gene transfer applications. PLoS Genetics 10: e169.
• Wilber, A.C., J.L. Frandsen, J.L. Geurts, D.A. Largaespada, P.B. Hackett, and R.S. McIvor. 2006. RNA as a source of transposase for Sleeping Beauty-mediated gene insertion and expression in somatic cells and tissues. Molecular Therapy 13: 625-630.
• Score, P.R., L. Belur, J.L. Frandsen, J.L. Geurts, P.B. Hackett, D.A. Largaespada, and R.S. McIvor. 2006. Sleeping Beauty-mediated transposition and long-term expression in vivo: application of the LoxP-Cre recombinase system for transposon-specific expression. Molecular Therapy, 13: 617-624.
• Huang, X, A.C. Wilber, L. Bao, D. Tuong, J. Tolar, P.J. Orchard, B.L. Levine, C.H. June, R.S. McIvor, B.R. Blazar, and X. Zhou. 2006. Stable gene transfer and expression in human primary T-cells by the Sleeping Beauty transposon system. Blood 107: 483-491.

Last modified on: August 10, 2009