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) 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 an active vertebrate transposon ("Sleeping Beauty") to viral and non-viral delivery systems for gene therapy.
Selected Publications (Pubmed Search)
Filareto, A., S. Parker, R. Darabi, L. Borges, T. Schaaf, T. Mayerhofer, J.S. Chamberlain, J.M. Ervasti, R.S. McIvor, M. Kyba and R.C.R. Perlingeiro. 2013. An ex vivo gene therapy approach to treat muscular dystrophy using iPS cells. Nature Communications 4: 1549.
Gori, J.L., B.C. Beard, N.P. Williams, C. Ironside, D. Swanson, R.S. McIvor, and H.P. Kiem. 2013. In vivo protection of activated Tyr22-dihydrofolate reductase gene-modified canine T lymphocytes from methotrexate. J Gene Med. 15: 233-241.
Aronovich, E.L., B.C. Hall, J.B. Bell, R.S. McIvor, and P.B. Hackett. 2013. Quantitative Analysis of α-L-Iduronidase Expression in Immunocompetent Mice Treated with the Sleeping Beauty Transposon System. PLoS One 8:e78161.
Moriarity B.S., E.P. Rahrmann, D.A. Beckmann, C.B. Conboy, A.L. Watson, D.F. Carlson, E.R. Olson, K.A. Hyland, S.C. Fahrenkrug, R.S. McIvor, and D.A. Largaespada. 2014. Simple and efficient methods for enrichment and isolation of endonuclease modified cells. PLoS One 9:e96114.
Schuster, D.J., J.A. Dykstra, M.S. Riedl, K.F. Kitto, L. Belur, R.S. McIvor, R.P. Elde, C.A. Fairbanks, and L. Vulchanova. 2014. Biodistribution of adeno-associated virus serotype 9 (AAV9) after intrathecal and intravenous delivery in mouse. Front Neuroanat 2014 8:42.
Schuster, D.J., L.R. Belur, M.S. Riedl, S.A. Schnell, K.M. Podetz-Pedersen, K.F. Kitto, R.S. McIvor, L. Vulchanova and C.A. Fairbanks. 2014. Supraspinal gene transfer by intrathecal adeno-associated virus serotype 5. Front Neuroanat 2014 8:66.
Podetz-Pedersen, K.M., V. Vezys, N.V. Somia, S.J. Russell and R.S. McIvor. 2014. Cellular immune response against firefly luciferase after Sleeping Beauty mediated gene transfer in vivo. Human Gene Therapy 25: 955-65.
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.
Hyland, K.A., E.R. Olson and R.S. McIvor. 2015. Sleeping Beauty-mediated drug resistance gene transfer in human hematopoietic progenitor cells. Human Gene Therapy, in press.