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Start Over Author "McIvor, R. Scott" Journal molecular therapy
38 results on '"McIvor, R. Scott"'

3. ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome

Author

Ou, Li, primary, DeKelver, Russell C., additional, Rohde, Michelle, additional, Tom, Susan, additional, Radeke, Robert, additional, St. Martin, Susan J., additional, Santiago, Yolanda, additional, Sproul, Scott, additional, Przybilla, Michael J., additional, Koniar, Brenda L., additional, Podetz-Pedersen, Kelly M., additional, Laoharawee, Kanut, additional, Cooksley, Renee D., additional, Meyer, Kathleen E., additional, Holmes, Michael C., additional, McIvor, R. Scott, additional, Wechsler, Thomas, additional, and Whitley, Chester B., additional

Published
2019
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4. Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing

Author

Laoharawee, Kanut, primary, DeKelver, Russell C., additional, Podetz-Pedersen, Kelly M., additional, Rohde, Michelle, additional, Sproul, Scott, additional, Nguyen, Hoang-Oanh, additional, Nguyen, Tam, additional, St. Martin, Susan J., additional, Ou, Li, additional, Tom, Susan, additional, Radeke, Robert, additional, Meyer, Kathleen E., additional, Holmes, Michael C., additional, Whitley, Chester B., additional, Wechsler, Thomas, additional, and McIvor, R. Scott, additional

Published
2018
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7. 439. Prolonged Expression of Secreted Enzymes in Dogs After Liver Delivery of Sleeping Beauty Transposons: Implications for Non-Viral Gene Therapy of Mucopolysaccharidoses Types I and VII

Author

Aronovich, Elena L., primary, Hyland, Kendra A., additional, Hall, Bryan C., additional, Bell, Jason B., additional, Olson, Erik R., additional, Rusten, Myra, additional, Hunter, David W., additional, Whitley, Chester B., additional, Ellinwood, N. Matthew, additional, McIvor, R. Scott, additional, and Hackett, Perry B., additional

Published
2016
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8. 485. ZFN-Mediated Liver-Targeting Gene Therapy Corrects Systemic and Neurological Disease of Mucopolysaccharidosis Type I

Author

Ou, Li, primary, DeKelver, Russell, additional, Tom, Susan, additional, Radeke, Robert, additional, Rohde, Michelle, additional, Manning-Bog, Amy, additional, Sproul, Scott, additional, Przybilla, Michael J., additional, Komar, Brenda L., additional, Podetz-Pedersen, Kelly, additional, Laoharawee, Kanut, additional, Cooksley, Renee D., additional, Holmes, Michael C., additional, Wechsler, Thomas, additional, McIvor, R. Scott, additional, and Whitley, Chester B., additional

Published
2016
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10. 484. In Vivo Zinc-Finger Nuclease Mediated Iduronate-2-Sulfatase (IDS) Target Gene Insertion and Correction of Metabolic Disease in a Mouse Model of Mucopolysaccharidosis Type II (MPS II)

Author

Laoharawee, Kanut, primary, DeKelver, Russell, additional, Podetz-Petersen, Kelly M., additional, Tom, Susan, additional, Radeke, Robert, additional, Rohde, Michelle, additional, Manning-Bog, Amy, additional, Sproul, Scott, additional, Ou, Li, additional, Meyer, Kathleen, additional, Holmes, Michael C., additional, Whitley, Chester B., additional, Wechsler, Thomas, additional, and McIvor, R. Scott, additional

Published
2016
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11. ZFN-Mediated In VivoGenome Editing Corrects Murine Hurler Syndrome

Author

Ou, Li, DeKelver, Russell C., Rohde, Michelle, Tom, Susan, Radeke, Robert, St. Martin, Susan J., Santiago, Yolanda, Sproul, Scott, Przybilla, Michael J., Koniar, Brenda L., Podetz-Pedersen, Kelly M., Laoharawee, Kanut, Cooksley, Renee D., Meyer, Kathleen E., Holmes, Michael C., McIvor, R. Scott, Wechsler, Thomas, and Whitley, Chester B.

Abstract

Mucopolysaccharidosis type I (MPS I) is a severe disease due to deficiency of the lysosomal hydrolase α-L-iduronidase (IDUA) and the subsequent accumulation of the glycosaminoglycans (GAG), leading to progressive, systemic disease and a shortened lifespan. Current treatment options consist of hematopoietic stem cell transplantation, which carries significant mortality and morbidity risk, and enzyme replacement therapy, which requires lifelong infusions of replacement enzyme; neither provides adequate therapy, even in combination. A novel in vivogenome-editing approach is described in the murine model of Hurler syndrome. A corrective copy of the IDUAgene is inserted at the albumin locus in hepatocytes, leading to sustained enzyme expression, secretion from the liver into circulation, and subsequent uptake systemically at levels sufficient for correction of metabolic disease (GAG substrate accumulation) and prevention of neurobehavioral deficits in MPS I mice. This study serves as a proof-of-concept for this platform-based approach that should be broadly applicable to the treatment of a wide array of monogenic diseases.

Published
2019
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12. Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In VivoGenome Editing

Author

Laoharawee, Kanut, DeKelver, Russell C., Podetz-Pedersen, Kelly M., Rohde, Michelle, Sproul, Scott, Nguyen, Hoang-Oanh, Nguyen, Tam, St. Martin, Susan J., Ou, Li, Tom, Susan, Radeke, Robert, Meyer, Kathleen E., Holmes, Michael C., Whitley, Chester B., Wechsler, Thomas, and McIvor, R. Scott

Abstract

Mucopolysaccharidosis type II (MPS II) is an X-linked recessive lysosomal disorder caused by deficiency of iduronate 2-sulfatase (IDS), leading to accumulation of glycosaminoglycans (GAGs) in tissues of affected individuals, progressive disease, and shortened lifespan. Currently available enzyme replacement therapy (ERT) requires lifelong infusions and does not provide neurologic benefit. We utilized a zinc finger nuclease (ZFN)-targeting system to mediate genome editing for insertion of the human IDS (hIDS) coding sequence into a “safe harbor” site, intron 1 of the albumin locus in hepatocytes of an MPS II mouse model. Three dose levels of recombinant AAV2/8 vectors encoding a pair of ZFNs and a hIDScDNA donor were administered systemically in MPS II mice. Supraphysiological, vector dose-dependent levels of IDS enzyme were observed in the circulation and peripheral organs of ZFN+donor-treated mice. GAG contents were markedly reduced in tissues from all ZFN+donor-treated groups. Surprisingly, we also demonstrate that ZFN-mediated genome editing prevented the development of neurocognitive deficit in young MPS II mice (6–9 weeks old) treated at high vector dose levels. We conclude that this ZFN-based platform for expression of therapeutic proteins from the albumin locus is a promising approach for treatment of MPS II and other lysosomal diseases.

Published
2018
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14. Systemic Correction of Storage Disease in MPS I NOD/SCID Mice Using the Sleeping Beauty Transposon System

Author

Aronovich, Elena L, primary, Bell, Jason B, additional, Khan, Shaukat A, additional, Belur, Lalitha R, additional, Gunther, Roland, additional, Koniar, Brenda, additional, Schachern, Patricia A, additional, Parker, Josh B, additional, Carlson, Cathy S, additional, Whitley, Chester B, additional, McIvor, R Scott, additional, Gupta, Pankaj, additional, and Hackett, Perry B, additional

Published
2009
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17. Erratum to “Sleeping Beauty-Mediated Transposition and Long-Term Expression in Vivo: Use of the LoxP/Cre Recombinase System to Distinguish Transposition-Specific Expression”

Author

Score, Paul R., primary, Belur, Lalitha R., additional, Frandsen, Joel L., additional, Geurts, Jennifer L., additional, Yamaguchi, Tomoyuki, additional, Somia, Nikunj V., additional, Hackett, Perry B., additional, Largaespada, David A., additional, and McIvor, R. Scott, additional

Published
2006
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24. 405. Correction of the Murine Model of Hereditary Tyrosinemia Type I Using Messenger RNA as a Source of Transposase for Sleeping Beauty Mediated Integration of the FAH Gene

Author

Wilber, Andrew, primary, Wangensteen, Kirk J., additional, Chen, Yixin, additional, Zhou, Lijuan, additional, Frandsen, Joel L., additional, Bell, Jason, additional, Chen, Zongyu, additional, Ekker, Stephen C., additional, McIvor, R. Scott, additional, and Wang, Xin, additional

Published
2006
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25. A picornaviral 2A-like sequence-based tricistronic vector allowing for high-level therapeutic gene expression coupled to a dual-reporter system

Author

Osborn, Mark J., primary, Panoskaltsis-Mortari, Angela, additional, McElmurry, Ron T., additional, Bell, Scott K., additional, Vignali, Dario A.A., additional, Ryan, Martin D., additional, Wilber, Andrew C., additional, McIvor, R. Scott, additional, Tolar, Jakub, additional, and Blazar, Bruce R., additional

Published
2005
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26. Real-Time in Vivo Imaging of Stem Cells Following Transgenesis by Transposition

Author

Tolar, Jakub, primary, Osborn, Mark, additional, Bell, Scott, additional, McElmurry, Ron, additional, Xia, Lily, additional, Riddle, Megan, additional, Panoskaltsis-Mortari, Angela, additional, Jiang, Yuehua, additional, McIvor, R. Scott, additional, Contag, Christopher H., additional, Yant, Stephen R., additional, Kay, Mark A., additional, Verfaillie, Catherine M., additional, and Blazar, Bruce R., additional

Published
2005
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28. 826. The Risk of Integration: Characterization and Testing of Cell Lines Engineered for the Study of Insertional Gene Activation

Author

McIvor, R. Scott, Somia, Nikunj V., and Converse, Andrea D.

Subjects
*CELL lines
Abstract

An abstract of the article "The Risk of Integration: Characterization and Testing of Cell Lines Engineered for the Study of Insertional Gene Activation," by R. Scott McIvor, Nikunj V. Somia and Andrea D. Converse is presented.

Published
2005
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29. Sleeping Beauty Transposon mediated Engineering of Human Primary T Cells for Therapy of CD19+ Lymphoid Malignancies.

Author

Xin Huang, Hongfeng Guo, Kang, Johnthomas, Choi, Suet, Zhou, Tom C., Tammana, Syam, Lees, Christopher J., Zhong Ze Li, Milone, Michael, Levine, Bruce L., Tolar, Jakub, June, Carl H., McIvor, R. Scott, Wagner, John E., Blazar, Bruce R., and Xianzheng Zhou

Subjects
*TRANSPOSONS, *T cells, *MOBILE genetic elements, *REPORTER genes, *GENE expression, *GENETIC engineering, *GENETIC recombination
Abstract

We have reported earlier that the non viral Sleeping Beauty (SB) transposon system can mediate genomic integration and long term reporter gene expression in human primary peripheral blood (PB) T cells. In order to test whether this system can be used for genetically modifying both PB T cells and umbilical cord blood (UCB) T cells as graft versus leukemia effector cells, an SB transposon was constructed to coexpress a single chain chimeric antigen receptor (CAR) for human CD19 and CD20. PB and UCB were nucleofected with the transposon and a transposase plasmid, activated and then expanded in culture using anti CD3/CD28 beads. Stable dual gene expression was confirmed in both T cell types, permitting enrichment by positive selection with Rituxan. The engineered CD4+ T cells and CD8+ T cells both exhibited specific cytotoxicity against CD19+ leukemia and lymphoma cell lines, as well as against CD19 transfectants, and produced high levels of antigen dependent Th1 (but not Th2) cytokines. The in vivo adoptive transfer of genetically engineered T cells significantly reduced tumor growth and prolonged the survival of the animal. Taken together, these data indicate that T cells from PB and UCB can be stably modified using a non viral DNA transfer system, and that such modified T cells may be useful in the treatment of refractory leukemia and lymphoma.Molecular Therapy (2008); 16 3, 580–589. doi:10.1038/sj.mt.6300404 [ABSTRACT FROM AUTHOR]

Published
2008
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30. Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human α-l-iduronidase gene

Author

Hartung, Seth D., Frandsen, Joel L., Pan, Dao, Koniar, Brenda L., Graupman, Patrick, Gunther, Roland, Low, Walter C., Whitley, Chester B., and McIvor, R. Scott

Subjects
*LYSOSOMAL storage diseases, *MUCOPOLYSACCHARIDOSIS, *GENES, *GENE therapy
Abstract

Murine models of lysosomal storage diseases provide an opportunity to evaluate the potential for gene therapy to prevent systemic manifestations of the disease. To determine the potential for treatment of mucopolysaccharidosis type I using a gene delivery approach, a recombinant adeno-associated virus (AAV) vector, vTRCA1, transducing the human iduronidase (IDUA) gene was constructed and 1 × 1010 particles were injected intravenously into 1-day-old Idua-/- mice. High levels of IDUA activity were present in the plasma of vTRCA1-treated animals that persisted for the 5-month duration of the study, with heart and lung of this group demonstrating the highest tissue levels of gene transfer and enzyme activity overall. vTRCA1-treated Idua-/- animals with measurable plasma IDUA activity exhibited histopathological evidence of reduced lysosomal storage in a number of tissues and were normalized with respect to urinary GAG excretion, craniofacial bony parameters, and body weight. In an open field test, vTRCA1-treated Idua-/- animals exhibited a significant reduction in total squares covered and a trend toward normalization in rearing events and grooming time compared to control-treated Idua-/- animals. We conclude that AAV-mediated transduction of the IDUA gene in newborn Idua-/- mice was sufficient to have a major curative impact on several of the most important parameters of the disease. [Copyright &y& Elsevier]

Published
2004
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32. 347. Long-Term Gene Expression and Phenotypic Correction of FANCC Deficient Lymphoblastoid Cells Using the Sleeping Beauty Transposon System

Author

Wadman, Shannon A., Clark, Karl J., Hackett, Perry B., McIvor, R Scott, and Essner, Jeffrey J.

Subjects
*GENE expression, *TRANSPOSONS
Abstract

An abstract of the article "Long-Term Gene Expression and Phenotypic Correction of FANCC Deficient Lymphoblastoid Cells Using the Sleeping Beauty Transposon System," by Shannon A. Wadman, Karl J. Clark, Perry B. Hackett, R Scott McIvor and Jeffrey J. Essner is presented.

Published
2005
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33. 1098. Duration of Expression of Sleeping Beauty Transposase by Hydrodynamic Injection of C57/BL6 Mice

Author

Bell, Jason B., Aronovich, Elena L., Larson-DeBruzzi, Beth E., Koniar, Brenda L., Gunther, Roland, Whitley, Chester B., McIvor, R. Scott, and Hackett, Perry B.

Subjects
*TRANSPOSONS, *GENE expression
Abstract

An abstract of the article "Duration of Expression of Sleeping Beauty Transposase by Hydrodynamic Injection of C57/BL6 Mice," by Jason B. Bell and colleagues is presented.

Published
2005
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34. 908. Evaluation of the Sleeping Beauty Transposon System in OTC Deficient Spf-Ash Mice: A Non-Viral Approach to a Cell Autonomous Metabolic Disease

Author

Wilber, Andy, Frandsen, Joel, Morizono, Hiroki, Tuchman, Mendel, and McIvor, R. Scott

Subjects
*METABOLIC disorders
Abstract

An abstract of the article "Evaluation of the Sleeping Beauty Transposon System in OTC Deficient Spf-Ash Mice: A Non-Viral Approach to a Cell Autonomous Metabolic Disease," by Andy Wilber and colleagues is presented.

Published
2005
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35. 338. Non-Viral Gene Transfer into Hematopoietic Cells of Artemis Deficient ScidA Mice Using the Sleeping BeautyTransposon System

Author

Wilber, Andy, Buckley, Shannon, Lakshmipathy, Uma, Frandsen, Joel, Cowan, Morton J., and McIvor, R. Scott

Subjects
*GENETIC transformation, *HEMATOPOIETIC growth factors
Abstract

An abstract of the article "Non-Viral Gene Transfer into Hematopoietic Cells of Artemis Deficient ScidA Mice Using the Sleeping Beauty Transposon System," by Andy Wilber, Shannon Buckley, Uma Lakshmipathy, Joel Frandsen, Morton J. Cowan and R. Scott McIvor is presented.

Published
2005
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36. 244. AAV Vector-Mediated Gene Therapy of Mucopolysaccharidosis Type I

Author

Belur, Lalitha, Lorenzo, Damaris, Aronovich, Elena, Low, Walter C., Whitley, Chester B., and McIvor, R. Scott

Subjects
*GENE therapy, *MUCOPOLYSACCHARIDOSIS I
Abstract

An abstract of the article "AAV Vector-Mediated Gene Therapy of Mucopolysaccharidosis Type I," by Lalitha Belur, Damaris Lorenzo, Elena Aronovich, Walter C. Low, Chester B. Whitley and R. Scott McIvor is presented.

Published
2005
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37. 193. Direct Comparison of Integration Efficiency and Stable Gene Expression Mediated by the Sleeping Beauty Transposon System and by the PhiC31 Phage Integrase System in Cultured Human Fibroblasts and in Various Stem Cell Targets

Author

Wilber, Andy, Montoya, Fernando, Geurts, Aron M., Largaespada, David A., Lakshmipathy, Uma, and McIvor, R. Scott

Subjects
*GENE expression, *STEM cells
Abstract

An abstract of the article "Direct Comparison of Integration Efficiency and Stable Gene Expression Mediated by the Sleeping Beauty Transposon System and by the PhiC31 Phage Integrase System in Cultured Human Fibroblasts and in Various Stem Cell Targets," by Andy Wilber, Fernando Montoya, Aron M. Geurts, David A. Largaespada, Uma Lakshmipathy and R. Scott McIvor is presented.

Published
2005
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38. 192. Non-Viral, Long-Term Gene Expression in Hematopoietic Cells Using the Sleeping Beauty Transposon System

Author

Liu, Linda N., Essner, Jeffrey J., Shivakumar, Rama, Li, Linhong, Wadman, Shannon A., Clark, Karl J., Nasevicious, Aidas, Fratantoni, Joseph C., McIvor, R. Scott, and Hackett, Perry B.

Subjects
*GENE expression, *HEMATOPOIETIC stem cells
Abstract

An abstract of the article "Non-Viral, Long-Term Gene Expression in Hematopoietic Cells Using the Sleeping Beauty Transposon System," by Linda N. Liu,1 Jeffrey J. Essner, Rama Shivakumar, Linhong Li, Shannon A. Wadman, Karl J. Clark, Aidas Nasevicious, Joseph C. Fratantoni, Scott McIvor and Perry B. Hackett is presented.

Published
2005
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