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.

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 mucopolysaccharidosis 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.