The general research interest of Dr. McIvor's laboratory is genetic therapy for inherited diseases and cancer. Specific research activities include: (i) Use of recombinant 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) 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; (iii) Adaptation of an active vertebrate transposon ("Sleeping Beauty") in the development non-viral genetic therapies; (ii) Chromosomal site directed correction of mutations in hematopoietic stem cells and other target tissues for the treatment of immunodeficiencies and other inherited disorders.
Selected Publications: PubMed Search
Ou, L., R.C. DeKelver, M. Rohde, S. Tom, R. Radeke, S. J. St. Martin, Y. Santiago, S. Sproul, M.J. Przybilla, B.L. Koniar, K.M. Podetz-Pedersen, K. Laoharawee, R.D. Cooksley, K.E. Meyer, M.C. Holmes, R.S. McIvor, T. Wechsler, and C.B. Whitley. 2019. ZFN-mediated in vivo genome editing corrects murine Hurler syndrome. Molecular Therapy 27:178-187.
Laoharawee K., R.C. DeKelver, K.M. Podetz-Pedersen, M. Rohde, S. Sproul, H.O. Nguyen, T. Nguyen, S.J. St. Martin, L. Ou, S. Tom, R. Radeke, K.E. Meyer, M.C. Holmes, C.B. Whitley, T. Weschler, and R.S. McIvor. 2018. Dose-dependent prevention of metabolic and neurologic disease in murine MPS II by ZFN-mediated in vivo genome editing. Molecular Therapy 26:1127-1136.
Aronovich, E.L., K.A. Hyland, B.C. Hall, J.B. Bell, E.R. Olson, M.U. Rusten, D.W. Hunter, N.M. Ellinwood, R.S. McIvor and P.B. Hackett. 2017. Prolonged expression of secreted enzymes in dogs after liver-directed delivery of Sleeping Beauty transposons: implications for non-viral gene therapy of systemic disease. Human Gene Therapy 28: 551-564.
Laoharawee, K., K.M. Podetz-Pedersen, T. Nguyen, L. Evenstar, K. Kitto, Z. Nan, C.A. Fairbanks, W.C. Low, K. Kozarsky, and R.S. McIvor. 2017.. Prevention of neurocognitive deficiency in mucopolysaccharidosis type II mice by CNS-directed, AAV9-mediated iduronate sulfatase gene transfer. Human Gene Therapy 28: 626-638.
Belur, L.R., A. Temme, K.M. Podetz-Pedersen, M. Riedl, L. Vulchanova, N. Robinson, L.R. Hanson, K. Kozarsky, W.H. Frey II, W.C. Low, and R.S. McIvor. 2017. Intranasal AAV mediated gene delivery and expression of human iduronidase in the CNS: A non-invasive and effective approach for prevention of neurologic disease in mucopolysacchardosis type I. Human Gene Therapy 28: 576-587.
Hyland, K.A., E.L. Aronovich, E.R. Olson, J.B. Bell, M. Urness, R. Gunther, D.W. Hunter, P.B. Hackett, and R.S. McIvor. 2017. Transgene expression in dogs after liver-directed hydrodynamic delivery of Sleeping Beauty transposons using balloon catheters. Human Gene Therapy 28: 541-550.
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. 2017. Lentivirus mediated correction of Artemis-deficient severe combined immunodeficiency. Human Gene Therapy 28: 112-124.
Podetz-Pedersen, K.M., E. R. Olson, N.V. Somia, S.J. Russell and 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.
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.