U of M: Department of Genetics, Cell Biology and Development

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	R. Scott McIvor, Ph.D. Professor
	Areas of Research: Selected Publications

Mailing Address: University of Minnesota Department of Genetics, Cell Biology, and Development 6-160 Jackson 321 Church St. SE Minneapolis, MN 55455 USA
Office: 5-102 MCB P: 612-626-1497 Email: mcivo001@umn.edu	Lab: 5-226 MCB P: 612-626-1435

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 cellsin 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.

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Selected Publications:

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. 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, in press.

Zayed, H., L. Xia, A. Yerich, S.R. Yant, M.A. Kay, M. Puttaraju, G. Mansfield, D.L. Wiest, R.S. McIvor, J. Tolar, B.R. Blazar. Correction of severe combined immune deficiency in multipotent adult progenitor cells by spliceosome-mediated RNA trans-splicing and sleeping beauty transposon delivery. Molecular Therapy, in press.

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. Messenger RNA as a Source of Transposase for Sleeping Beauty Transposon-mediated Correction of Hereditary Tyrosinemia Type I. Molecular Therapy, in press.

Tolar, J., A.J. Nauta, M.J. Osborn, A. Panoskaltsis-Mortari, R.T. McElmurry, S. Bell, L. Xia, N. Zhou, M. Riddle, T.M. Schroeder, J.J. Westendorf, R.S. McIvor, P.C.W. Hogendoom, K. Szuhai, L. Oseth, B. Hirsch, S.R. Yant, M.A. Kay, A. Peister, D.J. Prockop, W.E. Fibbe, and B.R Blazar. 2007. Sarcoma derived from cultured mesenchymal stem cells. Stem Cells 25: 371-379.

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.

Geurts A.M., A. Wilber, C.M. Carlson, P.D. Lobitz, K.J. Clark, P.B. Hackett, R.S. McIvor, and D.A. Largaespada. 2006. Conditional gene expression in the mouse using a Sleeping Beauty gene-trap transposon. BMC Biotechnol. 6:30

O’Brien, T.A., D.T. Tuong, L.M. Basso, R.S. McIvor, and P.J. Orchard. 2006. Co-expression of the uracil phosphoribosyltransferase gene with a chimeric human nerve growth factor receptor / cytosine deaminase fusion gene using a single retroviral vector augments cytotoxicity of transduced human T-cells exposed to 5-fluorocytosine. Human Gene Therapy, 17: 518-530.

Tolar J., M.J. O'Shaughnessy, A. Panoskaltsis-Mortari, R.T. McElmurry, S. Bell, M. Riddle, R.S. McIvor, S.R. Yant, M.A. Kay, D. Krause, C.M. Verfaillie, and B.R. Blazar. 2006. Host factors that impact the biodistribution and persistence of multipotent adult progenitor cells. Blood 107: 4182-4188.

Zayed, H., R.S. McIvor, D.L. Wiest, and B.R. Blazar. 2006. In vitro functional correction of the mutation responsible for murine severe combined immune deficiency by short fragment homologous recombination. Human Gene Therapy 17: 158-166.

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 Beautytransposon system. Blood107: 483-491.

Seth, G., R.S. McIvor, and W.-S. Hu. 2006. 17b-hydroxysteroid dehydrogenase type 7 (Hsd17b7) reverts cholesterol auxotrophy in NS0 cells. Journal of Biotechnology 121: 241-252.

Tolar J, MJ Osborn, S Bell, R McElmurry, L Xia, M Riddle, A Panoskaltsis-Mortari, Y Jiang, RS McIvor, CH Contag, SR Yant, MA Kay, CM Verfaillie, and BR Blazar (2005) Real time imaging of stem cells using transgenesis by transposition. Molecular Therapy 12:42-48.

Carlson, C.M., J.L. Frandsen, R.S. McIvor, and D.A. Largaespada. 2005. Somatic integration of an oncogene-harboring Sleeping Beauty transposon to model tumors in the mouse. Proceedings of the National Academy of Sciences USA 102: 17059-17064.

Wilber, A.C., J.L. Frandsen, K. Wangensteen, S.C. Ekker, X. Wang and R.S. McIvor. 2005. Dynamic gene expression following systemic delivery of plasmid DNA as determined by in vivobioluminescent imaging. Human Gene Therapy 16: 1325.

Zhang J.L., J. Cai, J.D. Jackson, C.A. Kuszynski, S. Walls, R.S. McIvor, and I.J. Fox. 2005. Long-term transgene expression and survival of transgene-expressing grafts following lentivirus transduction of bone marrow side population cells. Transplantation 79:882-888.

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 Therapeutics314: 668-674.

Osborn, M.J., A. Panoskaltsis-Mortari, R.T. McElmurry, S.K. Bell, D.A.A. Vignali, M.D. Ryan, A.C. Wilber, R.S. McIvor, J. Tolar, and B.R. Blazar. 2005. A picornaviral 2A-like sequence-based tricistronic vector allowing for high-level therapeutic gene expression coupled to a dual-reporter system. Molecular Therapy12:569-574.

Ohlfest, J.R, J.L. Frandsen, S.Fritz, P.D. Lobitz, S.G. Perkinson, K.J. Clark, G. Nelsestuen, N.S. Key, R.S. McIvor, P.B. Hackett and D.A. Largaespada. 2005. Phenotypic correction and long-term expression of factor VIII in hemophilic mice by immunotolerization and nonviral gene transfer using the Sleeping Beautytransposon system. Blood 105: 2691-2698.

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.

To view these and other publications visit http://www.ncbi.nlm.nih.gov/PubMed
search menu should say PubMed
type McIvor RS in the avaliable line

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