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Research Output 1978 2019

2019
4 Citations (Scopus)

ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome

Ou, L., DeKelver, R. C., Rohde, M., Tom, S., Radeke, R., St. Martin, S. J., Santiago, Y., Sproul, S., Przybilla, M., Koniar, B. L., Podetz-Pedersen, K. M., Laoharawee, K., Cooksley, R. D., Meyer, K. E., Holmes, M. C., Mc Ivor, R. S., Wechsler, T. & Whitley, C. B., Jan 2 2019, In : Molecular Therapy. 27, 1, p. 178-187 10 p.

Research output: Contribution to journalArticle

Open Access
Mucopolysaccharidosis I
Clinical Trials
Genome
Injections
Gene Editing
2018
18 Citations (Scopus)

Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing

Laoharawee, K., DeKelver, R. C., Podetz-Pedersen, K. M., Rohde, M., Sproul, S., Nguyen, H. O., Nguyen, T., St. Martin, S. J., Ou, L., Tom, S., Radeke, R., Meyer, K. E., Holmes, M. C., Whitley, C. B., Wechsler, T. & Mc Ivor, R. S., Apr 4 2018, In : Molecular Therapy. 26, 4, p. 1127-1136 10 p.

Research output: Contribution to journalArticle

Open Access
Mucopolysaccharidosis II
Zinc Fingers
Metabolic Diseases
Nervous System Diseases
Iduronate Sulfatase
2017
5 Citations (Scopus)
Open Access
Iduronidase
Mucopolysaccharidoses
Dependovirus
Nervous System Diseases
Central Nervous System
12 Citations (Scopus)

Lentivirus mediated correction of artemis-deficient severe combined immunodeficiency

Punwani, D., Kawahara, M., Yu, J., Sanford, U., Roy, S., Patel, K., Carbonaro, D. A., Karlen, A. D., Khan, S., Cornetta, K., Rothe, M., Schambach, A., Kohn, D. B., Malech, H. L., McIvor, R. S., Puck, J. M. & Cowan, M. J., Jan 1 2017, In : Human gene therapy. 28, 1, p. 112-124 13 p.

Research output: Contribution to journalArticle

Severe Combined Immunodeficiency
Lentivirus
B-Lymphocytes
Hematopoietic Stem Cells
T-Lymphocytes
12 Citations (Scopus)

Prevention of neurocognitive deficiency in mucopolysaccharidosis Type II mice by central nervous system-directed, AAV9-mediated iduronate sulfatase gene transfer

Laoharawee, K., Podetz-Pedersen, K. M., Nguyen, T. T., Evenstar, L. B., Kitto, K. F., Nan, Z., Fairbanks, C. A., Low, W. C., Kozarsky, K. F. & Mcivor, R. S., Aug 1 2017, In : Human gene therapy. 28, 8, p. 626-638 13 p.

Research output: Contribution to journalArticle

Iduronate Sulfatase
Mucopolysaccharidosis II
Central Nervous System
Genes
Glycosaminoglycans
4 Citations (Scopus)

Prolonged Expression of Secreted Enzymes in Dogs after Liver-Directed Delivery of Sleeping Beauty Transposons: Implications for Non-Viral Gene Therapy of Systemic Disease

Aronovich, E. L., Hyland, K. A., Hall, B. C., Bell, J. B., Olson, E. R., Rusten, M. U., Hunter, D. W., Matthew Ellinwood, N., Scott McIvor, R. & Hackett, P. B., Jul 1 2017, In : Human gene therapy. 28, 7, p. 551-564 14 p.

Research output: Contribution to journalArticle

Open Access
Beauty
Genetic Therapy
Dogs
Transgenes
Canidae
4 Citations (Scopus)

Transgene Expression in Dogs after Liver-Directed Hydrodynamic Delivery of Sleeping Beauty Transposons Using Balloon Catheters

Hyland, K. A., Aronovich, E. L., Olson, E. R., Bell, J. B., Rusten, M. U., Gunther, R., Hunter, D. W., Hackett, P. B. & Scott Mcivor, R., Jul 1 2017, In : Human Gene Therapy. 28, 7, p. 541-550 10 p.

Research output: Contribution to journalArticle

Open Access
Beauty
Hydrodynamics
Transgenes
Catheters
Dogs
2016
2 Citations (Scopus)
Transposases
Beauty
Hydrodynamics
Luciferases
DNA Transposable Elements
2015
16 Citations (Scopus)

Gene therapy for neurologic manifestations of mucopolysaccharidoses

Wolf, D. A., Banerjee, S., Hackett, P. B., Whitley, C. B., McIvor, R. S. & Low, W. C., Feb 1 2015, In : Expert Opinion on Drug Delivery. 12, 2, p. 283-296 14 p.

Research output: Contribution to journalReview article

Mucopolysaccharidoses
Neurologic Manifestations
Genetic Therapy
Nervous System
Brain
5 Citations (Scopus)

Role of transgene regulation in ex vivo lentiviral correction of artemis deficiency

Multhaup, M. M., Podetz-Pedersen, K. M., Karlen, A. D., Olson, E. R., Gunther, R., Somia, N. V., Blazar, B. R., Cowan, M. J. & McIvor, R. S., Apr 1 2015, In : Human gene therapy. 26, 4, p. 232-243 12 p.

Research output: Contribution to journalArticle

Transgenes
Phosphoglycerate Kinase
Peptide Elongation Factor 1
T-Lymphocytes
B-Lymphocytes

Sleeping beauty-mediated drug resistance gene transfer in human hematopoietic progenitor cells

Hyland, K. A., Olson, E. R. & Mcivor, R. S., Oct 2015, In : Human gene therapy. 26, 10, p. 657-663 7 p.

Research output: Contribution to journalArticle

Beauty
Hematopoietic Stem Cells
Drug Resistance
Methotrexate
Genes
2014
53 Citations (Scopus)
Dependovirus
Green Fluorescent Proteins
Central Nervous System
Afferent Neurons
Sensory Receptor Cells
12 Citations (Scopus)

Cellular immune response against firefly luciferase after sleeping beauty-mediated gene transfer in vivo

Podetz-Pedersen, K. M., Vezys, V., Somia, N. V., Russell, S. J. & Mc Ivor, R. S., Nov 1 2014, In : Human gene therapy. 25, 11, p. 955-965 11 p.

Research output: Contribution to journalArticle

Firefly Luciferases
Beauty
Luciferases
Cellular Immunity
Genes
12 Citations (Scopus)

Simple and efficient methods for enrichment and isolation of endonuclease modified cells

Moriarity, B. S., Rahrmann, E. P., Beckmann, D. A., Conboy, C. B., Watson, A. L., Carlson, D. F., Olson, E. R., Hyland, K. A., Fahrenkrug, S. C., Scott McIvor, R. & Largaespada, D. A., May 5 2014, In : PloS one. 9, 5, e96114.

Research output: Contribution to journalArticle

Endonucleases
Genes
Transcription
nucleases
cells
9 Citations (Scopus)

Supraspinal gene transfer by intrathecal adeno-associated virus serotype 5

Schuster, D. J., Belur, L. R., Riedl, M. S., Schnell, S. A., Podetz-Pedersen, K. M., Kitto, K. F., McIvor, R. S., Vulchanova, L. & Fairbanks, C. A., Aug 5 2014, In : Frontiers in Neuroanatomy. 8, AUG, 66.

Research output: Contribution to journalArticle

Dependovirus
Astrocytes
Neurons
Transgenes
Genes
2013
87 Citations (Scopus)

An ex vivo gene therapy approach to treat muscular dystrophy using inducible pluripotent stem cells

Filareto, A., Parker, S., Darabi, R., Borges, L., Iacovino, M., Schaaf, T., Mayerhofer, T., Chamberlain, J. S., Ervasti, J. M., McIvor, R. S., Kyba, M. & Perlingeiro, R. C. R., Apr 11 2013, In : Nature communications. 4, 1549.

Research output: Contribution to journalArticle

Utrophin
gene therapy
Gene therapy
Induced Pluripotent Stem Cells
Pluripotent Stem Cells
2 Citations (Scopus)

In vivo protection of activated tyr22-dihydrofolate reductase gene-modified canine T lymphocytes from methotrexate

Gori, J. L., Beard, B. C., Williams, N. P., Ironside, C., Swanson, D., Mc Ivor, R. S. & Kiem, H. P., Jun 1 2013, In : Journal of Gene Medicine. 15, 6-7, p. 233-241 9 p.

Research output: Contribution to journalArticle

Tetrahydrofolate Dehydrogenase
Methotrexate
Canidae
T-Lymphocytes
Genes
6 Citations (Scopus)

Oral contrast enhances the resolution of in-life NIS reporter gene imaging

Suksanpaisan, L., Pham, L., Mc Ivor, R. S., Russell, S. J. & Peng, K. W., Nov 1 2013, In : Cancer gene therapy. 20, 11, p. 638-641 4 p.

Research output: Contribution to journalArticle

Reporter Genes
Stomach
Barium Sulfate
Omentum
Gamma Rays
14 Citations (Scopus)

PR-b functionalized stealth liposomes for targeted delivery to metastatic colon cancer

Adil, M., Belur, L., Pearce, T. R., Levine, R. M., Tisdale, A. W., Sorenson, B. S., Mc Ivor, R. S. & Kokkoli, E., Apr 1 2013, In : Biomaterials Science. 1, 4, p. 393-401 9 p.

Research output: Contribution to journalArticle

Liposomes
DNA
Integrins
Polyethyleneimine
Genes
14 Citations (Scopus)
Iduronidase
Beauty
transposons
quantitative analysis
mucopolysaccharidosis
16 Citations (Scopus)
Green Fluorescent Proteins
Colon
Spinal Ganglia
Dependovirus
Neurons
2012
4 Citations (Scopus)

Increased longevity and metabolic correction following syngeneic BMT in a murine model of mucopolysaccharidosis type i

Wolf, D. A., Lenander, A. W., Nan, Z., Braunlin, E. A., Podetz-Pedersen, K. M., Whitley, C. B., Gupta, P., Low, W. C. & McIvor, R. S., Sep 1 2012, In : Bone marrow transplantation. 47, 9, p. 1235-1240 6 p.

Research output: Contribution to journalArticle

Iduronidase
Mucopolysaccharidoses
Mucopolysaccharidosis I
Glycosaminoglycans
Brain
31 Citations (Scopus)

Lysosomal enzyme can bypass the blood-brain barrier and reach the CNS following intranasal administration

Wolf, D. A., Hanson, L. R., Aronovich, E. L., Nan, Z., Low, W. C., Frey, W. H. & McIvor, R. S., May 1 2012, In : Molecular Genetics and Metabolism. 106, 1, p. 131-134 4 p.

Research output: Contribution to journalArticle

Iduronidase
Intranasal Administration
Blood-Brain Barrier
Enzymes
Viruses
2011
4 Citations (Scopus)

Characterization of the human artemis promoter by heterologous gene expression in vitro and in vivo

Multhaup, M. M., Gurram, S., Podetz-Pedersen, K. M., Karlen, A. D., Swanson, D. L., Somia, N. V., Hackett, P. B., Cowan, M. J. & Mc Ivor, R. S., Oct 1 2011, In : DNA and Cell Biology. 30, 10, p. 751-761 11 p.

Research output: Contribution to journalArticle

Green Fluorescent Proteins
Gene Expression
V(D)J Recombination
Genes
Peptide Elongation Factor 1
45 Citations (Scopus)

Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I

Wolf, D. A., Lenander, A. W., Nan, Z., Belur, L. R., Whitley, C. B., Gupta, P., Low, W. C. & McIvor, R. S., Jul 1 2011, In : Neurobiology of Disease. 43, 1, p. 123-133 11 p.

Research output: Contribution to journalArticle

Mucopolysaccharidoses
Mucopolysaccharidosis I
Iduronidase
Nervous System Diseases
Central Nervous System
43 Citations (Scopus)

Efficacy and safety of sleeping beauty transposon-mediated gene transfer in preclinical animal studies

Hackett, P. B., Aronovich, E. L., Hunter, D., Urness, M., Bell, J. B., Kass, S. J., Cooper, L. J. N. & Mc Ivor, R. S., Oct 1 2011, In : Current gene therapy. 11, 5, p. 341-349 9 p.

Research output: Contribution to journalArticle

Beauty
Hydrodynamics
Safety
Genes
Humulus
5 Citations (Scopus)

Efficient non-viral integration and stable gene expression in multipotent adult progenitor cells

Wilber, A., Ulloa Montoya, F., Hammer, L., Moriarity, B. S., Geurts, A. M., Largaespada, D. A., Verfaillie, C. M., Mc Ivor, R. S. & Lakshmipathy, U., Dec 1 2011, In : Stem Cells International. 717069.

Research output: Contribution to journalArticle

Transposases
Beauty
Stem Cells
Gene Expression
Clone Cells
28 Citations (Scopus)

Inhibition of angiogenesis and suppression of colorectal cancer metastatic to the liver using the Sleeping Beauty Transposon System

Belur, L., Podetz-Pedersen, K. M., Sorenson, B. S., Hsu, A. H., Parker, J. B., Carlson, C. S., Saltzman, D. A., Ramakrishnan, S. & Mc Ivor, R. S., Feb 10 2011, In : Molecular Cancer. 10, 14.

Research output: Contribution to journalArticle

Beauty
Colorectal Neoplasms
Plasmids
Hydroxymethylglutaryl-CoA Reductase Inhibitors
Liver
20 Citations (Scopus)

Sleeping Beauty-mediated correction of Fanconi anemia type C

Hyland, K. A., Olson, E. R., Clark, K. J., Aronovich, E. L., Hackett, P. B., Blazar, B. R., Tolar, J. & Scott Mcivor, R., Sep 1 2011, In : Journal of Gene Medicine. 13, 9, p. 462-469 8 p.

Research output: Contribution to journalArticle

Fanconi Anemia
Beauty
Mitomycin
Electroporation
Green Fluorescent Proteins

Sleeping Beauty Transposon-Mediated Stable Gene Delivery

Wilber, A., Zhou, X., Huang, X., Kaufman, D. S. & Mc Ivor, R. S., Oct 25 2011, Primary and Stem Cells: Gene Transfer Technologies and Applications. John Wiley and Sons, p. 135-161 27 p.

Research output: Chapter in Book/Report/Conference proceedingChapter

Beauty
Genes
16 Citations (Scopus)

Therapeutic delivery of mRNA: The medium is the message

Mc Ivor, R. S., May 1 2011, In : Molecular Therapy. 19, 5, p. 822-823 2 p.

Research output: Contribution to journalComment/debate

Gene Transfer Techniques
Toll-Like Receptors
RNA Interference
Innate Immunity
Gene Expression
88 Citations (Scopus)
Beauty
Genetic Therapy
Mucopolysaccharidosis VII
Mucopolysaccharidosis I
DNA Viruses
2010
20 Citations (Scopus)

Cytotoxicity associated with artemis overexpression after lentiviral vector-mediated gene transfer

Multhaup, M., Karlen, A. D., Swanson, D. L., Wilber, A., Somia, N. V., Cowan, M. J. & Mc Ivor, R. S., Jul 1 2010, In : Human Gene Therapy. 21, 7, p. 865-875 11 p.

Research output: Contribution to journalArticle

Severe Combined Immunodeficiency
Cell Survival
V(D)J Recombination
Phosphoglycerate Kinase
Genes
61 Citations (Scopus)
Dependovirus
Spinal Puncture
Sensory Receptor Cells
Green Fluorescent Proteins
Genes
27 Citations (Scopus)

Gene expression in lung and liver after intravenous infusion of polyethylenimine complexes of Sleeping Beauty transposons

Podetz-Pedersen, K. M., Bell, J. B., Steele, T. W. J., Wilber, A., Shier, W. T., Belur, L., Mc Ivor, R. S. & Hackett, P. B., Feb 1 2010, In : Human gene therapy. 21, 2, p. 210-220 11 p.

Research output: Contribution to journalArticle

Polyethyleneimine
Beauty
Intravenous Infusions
Gene Expression
Lung
8 Citations (Scopus)

In vivo selection of human embryonic stem cell-derived cells expressing methotrexate-resistant dihydrofolate reductase

Gori, J. L., Tian, X., Swanson, D., Gunther, R., Shultz, L. D., Mc Ivor, R. S. & Kaufman, D. S., Feb 1 2010, In : Gene therapy. 17, 2, p. 238-249 12 p.

Research output: Contribution to journalArticle

Tetrahydrofolate Dehydrogenase
Methotrexate
Genetic Therapy
Teratoma
Cell- and Tissue-Based Therapy
Tetrahydrofolate Dehydrogenase
Stem cells
Methotrexate
Cells
Gene therapy
2009
17 Citations (Scopus)

DNA transposons for modification of human primary T lymphocytes.

Huang, X., Wilber, A., McIvor, R. S. & Zhou, X., 2009, In : Methods in molecular biology (Clifton, N.J.). 506, p. 115-126 12 p.

Research output: Contribution to journalArticle

DNA Transposable Elements
T-Lymphocytes
Genes
HIV-1
X-Linked Combined Immunodeficiency Diseases
5 Citations (Scopus)

High-titer lentiviral vectors stimulate fetal calf serum-specific human CD4 T-cell responses: Implications in human gene therapy

Bao, L., Guo, H., Huang, X., Tammana, S., Wong, M., Mc Ivor, R. S. & Zhou, X., Apr 3 2009, In : Gene therapy. 16, 6, p. 788-795 8 p.

Research output: Contribution to journalArticle

Genetic Therapy
T-Lymphocytes
Serum
Hemagglutinins
Dendritic Cells

Sleeping Beauty-Mediated Transposition in Stem Cells

Wilber, A., Tolar, J., Blazar, B. R., Verfaillie, C. M., Lakshmipathy, U., Kaufman, D. S. & McIvor, R. S., Apr 17 2009, Emerging Technology Platforms for Stem Cells. John Wiley & Sons, Inc., p. 341-362 22 p.

Research output: Chapter in Book/Report/Conference proceedingChapter

Beauty
Stem cells
Stem Cells
58 Citations (Scopus)

Sleeping beauty transposition from nonintegrating lentivirus

Vink, C. A., Gaspar, H. B., Gabriel, R., Schmidt, M., Mc Ivor, R. S., Thrasher, A. J. & Qasim, W., May 6 2009, In : Molecular Therapy. 17, 7, p. 1197-1204 8 p.

Research output: Contribution to journalArticle

Beauty
Lentivirus
Transposases
Integrases
HIV-1
88 Citations (Scopus)

Stable gene transfer and expression in cord blood-derived CD34+ hematopoietic stem and progenitor cells by a hyperactive Sleeping Beauty transposon system

Xue, X., Huang, X., Nodland, S. E., Mátés, L., Ma, L., Izsvák, Z., Ivics, Z., LeBien, T. W., McIvor, R. S., Wagner, J. E. & Zhou, X., Nov 19 2009, In : Blood. 114, 7, p. 1319-1330 12 p.

Research output: Contribution to journalArticle

Gene transfer
Beauty
Hematopoietic Stem Cells
Fetal Blood
Gene expression
66 Citations (Scopus)

Systemic correction of storage disease in MPS I NOD/SCID mice using the sleeping beauty transposon system

Aronovich, E. L., Bell, J. B., Khan, S. A., Belur, L., Gunther, R., Koniar, B., Schachern, P. A., Parker, J. B., Carlson, C. S., Whitley, C. B., Mc Ivor, R. S., Gupta, P. & Hackett, P. B., Apr 22 2009, In : Molecular Therapy. 17, 7, p. 1136-1144 9 p.

Research output: Contribution to journalArticle

Mucopolysaccharidosis I
Beauty
Severe Combined Immunodeficiency
Transposases
Glycosaminoglycans
2008
5 Citations (Scopus)
Receptor, Fibroblast Growth Factor, Type 1
Dependovirus
Purkinje Cells
Cerebellar Diseases
Heparan Sulfate Proteoglycans
29 Citations (Scopus)

A facile method for somatic, lifelong manipulation of multiple genes in the mouse liver

Wangensteen, K. J., Wilber, A., Keng, V. W., He, Z., Matise, I., Wangensteen, L., Carson, C. M., Chen, Y., Steer, C. J., Mc Ivor, R. S., Largaespada, D. A., Wang, X. & Ekker, S. C., May 1 2008, In : Hepatology. 47, 5, p. 1714-1724 11 p.

Research output: Contribution to journalArticle

Hydrolases
Hepatocytes
Liver
Genes
Gene Expression
7 Citations (Scopus)
Spinocerebellar Ataxias
Trinucleotide Repeats
Antisense RNA
Gene therapy
Molecular Models
14 Citations (Scopus)

Liver-directed gene therapy using the sleeping beauty transposon system

Belur, L., Mc Ivor, R. S. & Wilber, A., Sep 16 2008, Gene Therapy Protocols: Design and Characterization of Gene Transfer Vectors. Humana Press, Vol. 434. p. 267-276 10 p. (Methods in Molecular Biology; vol. 434).

Research output: Chapter in Book/Report/Conference proceedingChapter

Beauty
Genetic Therapy
Liver
Animal Disease Models
Genetically Modified Animals
93 Citations (Scopus)

Sleeping beauty transposon-mediated engineering of human primary T cells for therapy of CD19+ lymphoid malignancies

Huang, X., Guo, H., Kang, J., Choi, S., Zhou, T. C., Tammana, S., Lees, C. J., Li, Z. Z., Milone, M., Levine, B. L., Tolar, J., June, C. H., McIvor, R. S., Wagner, J. E., Blazar, B. R. & Zhou, X., Jan 1 2008, In : Molecular Therapy. 16, 3, p. 580-589 10 p.

Research output: Contribution to journalArticle

Beauty
Human Engineering
Cell- and Tissue-Based Therapy
T-Lymphocytes
Neoplasms
2007
17 Citations (Scopus)

Correction of DNA protein kinase deficiency by spliceosome-mediated RNA trans-splicing and sleeping beauty transposon delivery

Zayed, H., Xia, L., Yerich, A., Yant, S. R., Kay, M. A., Puttaraju, M., McGarrity, G. J., Wiest, D. L., McIvor, R. S., Tolar, J. & Blazar, B. R., Jul 1 2007, In : Molecular Therapy. 15, 7, p. 1273-1279 7 p.

Research output: Contribution to journalArticle

Trans-Splicing
Polynucleotide 5'-Hydroxyl-Kinase
Spliceosomes
Beauty
Protein Deficiency
82 Citations (Scopus)

Efficient and stable transgene expression in human embryonic stem cells using transposon-mediated gene transfer

Wilber, A., Linehan, J. L., Tian, X., Woll, P. S., Morris, J. K., Belur, L., Mc Ivor, R. S. & Kaufman, D. S., Nov 1 2007, In : STEM CELLS. 25, 11, p. 2919-2927 9 p.

Research output: Contribution to journalArticle

Transgenes
Beauty
Transposases
Genes
RNA